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10-Q – 2026-05-05 – bmrn-20260331.htm
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Commission File Number: 000-26727 ______________________________________ BioMarin Pharmaceutical Inc . (Exact name of registrant as specified in its charter) ______________________________________ Delaware 68-0397820 (State or other jurisdiction of incorporation or organization) (I.R.S. Employer Identification No.) 770 Lindaro Street San Rafael California 94901 (Address of principal executive offices) (Zip Code) ( 415 ) 506-6700 (Registrant’s telephone number including area code) ______________________________________ Securities registered pursuant to Section 12(b) of the Act: Title of each class Trading Symbol(s) Name of each exchange on which registered Common Stock, par value $0.001 BMRN The Nasdaq Global Select Market ______________________________________ Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days. Yes ☒ No ☐ Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T (§ 232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files). Yes ☒ No ☐ Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company, or an emerging growth company. See the definitions of “large accelerated filer,” “accelerated filer,” “smaller reporting company,” and “emerging growth company” in Rule 12b-2 of the Exchange Act. Large Accelerated Filer ☒ Accelerated Filer ☐ Non-accelerated Filer ☐ Smaller Reporting Company ☐ Emerging Growth Company ☐ If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐ Table of Contents Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act.) Yes ☐ No ☒ Applicable only to corporate issuers: Indicate the number of shares outstanding of each of the issuer’s classes of common stock, as of the latest practicable date: 193,284,438 shares of common stock, par value $0.001, outstanding as of April 23, 2026. Table of Contents Unless the context suggests otherwise, references in this Quarterly Report on Form 10-Q to “BioMarin,” the “Company,” “we,” “us,” and “our” refer to BioMarin Pharmaceutical Inc. and, where appropriate, its wholly owned subsidiaries. BioMarin ® , BRINEURA ® , GALAFOLD ® , KUVAN ® , NAGLAZYME ® , PALYNZIQ ® , POMBILITI ® + OPFOLDA ® , ROCTAVIAN ® , VIMIZIM ® and VOXZOGO ® are our registered trademarks. ALDURAZYME ® is a registered trademark of BioMarin/Genzyme LLC. All other brand names and service marks, trademarks and other trade names appearing in this report are the property of their respective owners. Forward-Looking Statements This Quarterly Report on Form 10-Q contains “forward-looking statements” as defined under securities laws. Many of these statements can be identified by the use of terminology such as “believes,” “expects,” “intends,” “anticipates,” “plans,” “may,” “will,” “could,” “would,” “projects,” “continues,” “estimates,” “potential,” “opportunity” or the negative versions of these terms and other similar expressions. You should not place undue reliance on these types of forward-looking statements, which speak only as of the date that they were made. These forward-looking statements are based on the beliefs and assumptions of our management based on information currently available to management and should be considered in connection with any written or oral forward-looking statements that we may issue in the future as well as other cautionary statements we have made and may make. Our actual results or experience could differ significantly from the forward-looking statements. Factors that could cause or contribute to these differences include those discussed in “Risk Factors,” in Part II, Item 1A of this Quarterly Report on Form 10-Q as well as information provided elsewhere in this Quarterly Report on Form 10-Q and our Annual Report on Form 10-K for the year ended December 31, 2025, which was filed with the Securities and Exchange Commission (the SEC) on February 26, 2026. You should carefully consider that information before you make an investment decision. Moreover, we operate in a very competitive and rapidly changing environment. New risks emerge from time to time. It is not possible for our management to predict all risks, nor can we assess the impact of all factors on our business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements we make. In light of these risks, uncertainties and assumptions, the forward-looking events and circumstances discussed in this Quarterly Report on Form 10-Q may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. Except as required by law, we do not undertake any obligation to release publicly any revisions to these forward-looking statements after completion of the filing of this Quarterly Report on Form 10-Q to reflect later events or circumstances or the occurrence of unanticipated events. The discussion of the Company’s financial condition and results of operations should be read in conjunction with the Company’s Condensed Consolidated Financial Statements and the related Notes thereto included in this Quarterly Report on Form 10-Q. Table of Contents Risk Factors Summary The following is a summary of the principal risks that could adversely affect our business, financial condition, operating results, cash flows or stock price. Discussion of the risks listed below, and other risks that we face, are discussed in the section titled “Risk Factors” in Part II, Item 1A of this Quarterly Report on Form 10-Q. Business and Operational Risks • Our success depends on our ability to manage our growth and execute our corporate strategy. • If we fail to develop new products and product candidates or compete successfully with respect to acquisitions, joint ventures, licenses or other collaboration opportunities, our ability to continue to expand our product pipeline and our growth and development would be impaired. • We have in the past and may in the future pursue acquisitions of other companies or businesses, which could divert our management's attention, fail to achieve the anticipated benefits and/or expose us to other risks or difficulties. • If we do not achieve our projected development goals in the timeframes we announce or fail to achieve such goals, the commercialization of our product candidates may be delayed or never occur and the credibility of our management may be adversely affected and, as a result, our stock price may decline. • If we fail to compete successfully with respect to product sales, we may be unable to generate sufficient sales to recover our expenses related to the development of a product program or to justify continued marketing of a product and our revenues could be adversely affected. • If we fail to obtain and maintain an adequate level of coverage and reimbursement for our products by third-party payers, the sales of our products would be adversely affected or there may be no commercially viable markets for our products. • Because the target patient populations for our products are relatively small, we must achieve significant market share and maintain high per-patient prices for our products to achieve and maintain profitability. • Changes in methods of treatment of disease or failure of our products to gain acceptance by patients or the medical community could negatively impact demand for our products and adversely affect revenues. Risks Related to the Amicus Acquisition • We may not realize the anticipated benefits from the Amicus Acquisition or accurately forecast the future performance of the combined company. • We have incurred and expect to incur material expenses related to the Amicus Acquisition. • We may not realize the anticipated cost savings from the Amicus Acquisition. Regulatory Risks • If we fail to obtain regulatory approval to commercially market and sell our product candidates, or if approval of our product candidates is delayed, we will be unable to generate revenues from the sale of these product candidates, our potential for generating positive cash flow will be diminished, and the capital necessary to fund our operations will increase. • Any product for which we have obtained regulatory approval, or for which we obtain approval in the future, is subject to, or will be subject to, extensive ongoing regulatory requirements by the U.S. Food and Drug Administration (FDA), the European Commission (EC), the European Medicines Agency (EMA) and other comparable international regulatory authorities, and if we fail to comply with regulatory requirements or if we experience unanticipated problems with our products, we may be subject to penalties, we will be unable to generate revenues from the sale of such products, our potential for generating positive cash flow will be diminished, and the capital necessary to fund our operations will be increased. • To obtain regulatory approval to market our products, preclinical studies and costly and lengthy clinical trials are required and the results of the studies and trials are highly uncertain. Likewise, preliminary, initial or interim data from clinical trials should be considered carefully and with caution because the final data may be materially different from the preliminary, initial or interim data, particularly as more patient data become available. • Government price controls or other changes in pricing regulation could restrict the amount that we are able to charge for our current and future products, which would adversely affect our revenues and results of operations. • Government healthcare reform could increase our costs and adversely affect our revenues and results of operations. Financial and Financing Risks Table of Contents • If we fail to obtain the capital necessary to fund our operations, our financial results and financial condition will be adversely affected and we will have to delay or terminate some or all of our product development programs. • We have incurred in the past and may in the future incur substantial indebtedness that may decrease our business flexibility, access to capital, and/or increase our borrowing costs, which may adversely affect our operations and financial results. Manufacturing Risks • If we fail to comply with manufacturing regulations, our financial results and financial condition will be adversely affected. • If we are unable to successfully develop and maintain manufacturing processes for our product candidates to produce sufficient quantities at acceptable costs, we may be unable to support a clinical trial or be forced to terminate a program, or if we are unable to produce sufficient quantities of our products at acceptable costs, we may be unable to meet commercial demand, lose potential revenue, have reduced margins or be forced to terminate a program. • Supply interruptions may disrupt our inventory levels and the availability of our products and product candidates and cause delays in obtaining regulatory approval for our product candidates, or harm our business by reducing our revenues. Risks Related to International Operations • We conduct a significant amount of our operations and generate a significant percentage of our sales outside of the U.S., which subjects us to additional business risks that could adversely affect our revenues and results of operations. • A significant portion of our international sales are made based on special access programs, and changes to these programs could adversely affect our product sales and revenues in these countries. • Our international operations pose currency risks, which may adversely affect our operating results and net income. Intellectual Property Risks • If we are unable to protect our intellectual property, we may not be able to compete effectively or preserve our market shares. • Competitors and other third parties may have developed intellectual property that could limit our ability to market and commercialize our products and product candidates, if approved. Table of Contents BIOMARIN PHARMACEUTICAL INC. TABLE OF CONTENTS Page PART I. FINANCIAL INFORMATION 3 Item 1. Financial Statements 3 Condensed Consolidated Statements of Comprehensive Income (Unaudited) for the three months ended March 31, 2026 and 2025 3 Condensed Consolidated Balance Sheets as of March 31, 2026 (Unaudited) and December 31, 2025 4 Condensed Consolidated Statement of Stockholders’ Equity (Unaudited) for the three months ended March 31, 2026 and 2025 5 Condensed Consolidated Statements of Cash Flows (Unaudited) for the three months ended March 31, 2026 and 2025 6 Notes to Condensed Consolidated Financial Statements (Unaudited) 7 Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations 19 Item 3. Quantitative and Qualitative Disclosures about Market Risk 30 Item 4. Controls and Procedures 30 PART II. OTHER INFORMATION 31 Item 1. Legal Proceedings 31 Item 1A. Risk Factors 31 Item 2. Unregistered Sales of Equity Securities and Use of Proceeds 64 Item 3. Defaults Upon Senior Securities 64 Item 4. Mine Safety Disclosures 64 Item 5. Other Information 64 Item 6. Exhibits 65 SIGNATURES 67 2 PART I. FINANCIAL INFORMATION Item 1. Financial Statements BIOMARIN PHARMACEUTICAL INC. CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE INCOME Three Months Ended March 31, 2026 and 2025 (In thousands, except per share amounts) (unaudited) Three Months Ended March 31, 2026 2025 REVENUES: Net product revenues $ 760,078 $ 734,644 Royalty and other revenues 6,130 10,501 Total revenues 766,208 745,145 OPERATING EXPENSES: Cost of sales 194,999 151,558 Research and development 178,796 158,731 Selling, general and administrative 258,290 206,116 Intangible asset amortization 4,483 4,847 Total operating expenses 636,568 521,252 INCOME FROM OPERATIONS 129,640 223,893 Interest income 22,560 19,013 Interest expense ( 14,958 ) ( 2,863 ) Other income (expense), net 3,961 ( 1,954 ) INCOME BEFORE INCOME TAXES 141,203 238,089 Provision for income taxes 35,676 52,403 NET INCOME $ 105,527 $ 185,686 EARNINGS PER SHARE, BASIC $ 0.55 $ 0.97 EARNINGS PER SHARE, DILUTED $ 0.54 $ 0.95 Weighted average common shares outstanding, basic 192,497 190,967 Weighted average common shares outstanding, diluted 197,671 196,474 COMPREHENSIVE INCOME $ 114,763 $ 143,184 The accompanying notes are an integral part of these Condensed Consolidated Financial Statements. 3 BIOMARIN PHARMACEUTICAL INC. CONDENSED CONSOLIDATED BALANCE SHEETS March 31, 2026 and December 31, 2025 (In thousands, except share amounts) March 31, 2026 December 31, 2025 ⁽¹⁾ ASSETS (unaudited) Current assets: Cash and cash equivalents $ 2,222,435 $ 1,311,679 Short-term investments — 248,930 Accounts receivable, net 903,914 908,214 Inventory 1,273,221 1,298,883 Other current assets 205,500 185,784 Total current assets 4,605,070 3,953,490 Noncurrent assets: Long-term investments — 492,242 Property, plant and equipment, net 958,071 952,508 Intangible assets, net 204,662 213,837 Goodwill 196,199 196,199 Deferred tax assets 1,500,598 1,508,697 Restricted cash equivalents 850,000 — Other assets 276,416 277,049 Total assets $ 8,591,016 $ 7,594,022 LIABILITIES AND STOCKHOLDERS’ EQUITY Current liabilities: Accounts payable and accrued liabilities $ 793,152 $ 759,031 Total current liabilities 793,152 759,031 Noncurrent liabilities: Long-term debt, net 1,430,282 597,176 Other long-term liabilities 155,475 150,816 Total liabilities 2,378,909 1,507,023 Stockholders’ equity: Common stock, $ 0.001 par value: 500,000,000 shares authorized; 193,268,870 and 192,300,101 shares issued and outstanding, respectively 193 192 Additional paid-in capital 5,966,868 5,956,582 Company common stock held by the Nonqualified Deferred Compensation Plan ( 10,450 ) ( 10,508 ) Accumulated other comprehensive loss ( 4,237 ) ( 13,473 ) Retained earnings 259,733 154,206 Total stockholders’ equity 6,212,107 6,086,999 Total liabilities and stockholders’ equity $ 8,591,016 $ 7,594,022 (1) December 31, 2025 balances were derived from the audited Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025, filed with the SEC on February 26, 2026. The accompanying notes are an integral part of these Condensed Consolidated Financial Statements. 4 BIOMARIN PHARMACEUTICAL INC. CONDENSED CONSOLIDATED STATEMENTS OF STOCKHOLDERS’ EQUITY Three Months Ended March 31, 2026 and 2025 (In thousands) (unaudited) Three Months Ended March 31, 2026 2025 Shares of common stock, beginning balances (1) 192,300 190,761 Issuances under equity incentive plans 969 995 Shares of common stock, ending balances 193,269 191,756 Total stockholders' equity, beginning balances (1) $ 6,086,999 $ 5,657,990 Common stock: Beginning balances (1) 192 191 Issuances under equity incentive plans, net of tax 1 1 Ending balances 193 192 Additional paid-in capital: Beginning balances (1) 5,956,582 5,802,068 Issuances under equity incentive plans, net of tax ( 37,668 ) ( 49,333 ) Stock-based compensation 48,012 41,617 Change in Common stock held by the Nonqualified Deferred Compensation plan (NQDC) ( 58 ) ( 50 ) Ending balances 5,966,868 5,794,302 Company common stock held by the NQDC: Beginning balances (1) ( 10,508 ) ( 11,227 ) Common stock held by the NQDC 58 50 Ending balances ( 10,450 ) ( 11,177 ) Accumulated other comprehensive income (loss): Beginning balances (1) ( 13,473 ) 61,653 Other comprehensive income (loss) 9,236 ( 42,502 ) Ending balances ( 4,237 ) 19,151 Retained earnings (accumulated deficit) Beginning balances (1) 154,206 ( 194,695 ) Net income 105,527 185,686 Ending balances 259,733 ( 9,009 ) Total stockholders' equity, ending balances $ 6,212,107 $ 5,793,459 (1) The beginning balances for the three-month periods were derived from the audited Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025 filed with the SEC on February 26, 2026. The accompanying notes are an integral part of these Condensed Consolidated Financial Statements. 5 BIOMARIN PHARMACEUTICAL INC. CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS Three Months Ended March 31, 2026 and 2025 (In thousands) (unaudited) Three Months Ended March 31, 2026 2025 CASH FLOWS FROM OPERATING ACTIVITIES: Net income $ 105,527 $ 185,686 Adjustments to reconcile net income to net cash provided by operating activities: Depreciation and amortization 16,411 22,069 Non-cash interest expense 6,086 660 Accretion of discount on investments ( 455 ) ( 1,362 ) Stock-based compensation 43,458 37,700 Impairment of assets — 2,967 Deferred income taxes 9,220 28,429 Unrealized foreign exchange losses (gains) 6,710 ( 10,026 ) Other ( 5,374 ) ( 1,267 ) Changes in operating assets and liabilities: Accounts receivable, net ( 7,159 ) ( 57,590 ) Inventory 44,490 ( 24,335 ) Other current assets ( 11,551 ) ( 6,327 ) Other assets 1,484 ( 1,624 ) Accounts payable and accrued liabilities 3,100 ( 2,655 ) Other long-term liabilities 8,704 2,069 Net cash provided by operating activities 220,651 174,394 CASH FLOWS FROM INVESTING ACTIVITIES: Purchases of property, plant and equipment ( 20,923 ) ( 16,768 ) Maturities and sales of investments 767,277 77,804 Purchases of investments ( 25,792 ) ( 89,274 ) Other 4,966 — Net cash provided by (used in) investing activities 725,528 ( 28,238 ) CASH FLOWS FROM FINANCING ACTIVITIES: Taxes paid related to net share settlement of equity awards ( 28,180 ) ( 38,779 ) Proceeds from issuance of debt 850,000 — Payments of debt issuance costs ( 8,653 ) — Net cash provided by (used in) financing activities 813,167 ( 38,779 ) Effect of exchange rate changes on cash 1,410 ( 1,416 ) NET INCREASE IN CASH, CASH EQUIVALENTS AND RESTRICTED CASH EQUIVALENTS 1,760,756 105,961 Cash, cash equivalents and restricted cash equivalents: Beginning of period $ 1,311,679 $ 942,842 End of period $ 3,072,435 $ 1,048,803 SUPPLEMENTAL CASH FLOW DISCLOSURES: Cash paid for income taxes $ 14,644 $ 10,388 SUPPLEMENTAL CASH FLOW DISCLOSURES FOR NON-CASH INVESTING AND FINANCING ACTIVITIES: Accounts payable and accrued liabilities related to fixed assets $ 11,200 $ 4,470 Accounts payable and accrued liabilities related to intangible assets $ 4,481 $ — The accompanying notes are an integral part of these Condensed Consolidated Financial Statements. 6 Table of Contents BIOMARIN PHARMACEUTICAL INC. NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS (UNAUDITED) (In thousands of U.S. Dollars, except per share amounts or as otherwise disclosed) (1) BUSINESS OVERVIEW AND SIGNIFICANT ACCOUNTING POLICIES Nature of Operations BioMarin Pharmaceutical Inc. (the Company or BioMarin) is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with a portfolio of commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, the Company seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. Basis of Presentation These Condensed Consolidated Financial Statements have been prepared pursuant to U.S. generally accepted accounting principles (U.S. GAAP) and the rules and regulations of the Securities and Exchange Commission for Quarterly Reports on Form 10-Q and do not include all of the information and note disclosures required by U.S. GAAP for complete financial statements, although management believes that the disclosures herein are adequate to ensure that the information presented is not misleading. The Condensed Consolidated Financial Statements should therefore be read in conjunction with the Consolidated Financial Statements and Notes thereto for the fiscal year ended December 31, 2025 included in the Company’s Annual Report on Form 10-K. The Condensed Consolidated Financial Statements include the accounts of the Company and its wholly owned subsidiaries. All intercompany transactions have been eliminated. The results of operations for the three months ended March 31, 2026 are not necessarily indicative of the results that may be expected for the fiscal year ending December 31, 2026 or any other period. Management performed an evaluation of the Company’s activities through the date of filing of this Quarterly Report on Form 10-Q and has concluded that there were no subsequent events or transactions that occurred subsequent to the balance sheet date and prior to filing this Quarterly Report on Form 10-Q that would require recognition or disclosure in the Condensed Consolidated Financial Statements, except for the transactions disclosed in Note 1 3 to these Condensed Consolidated Financial Statements. Use of Estimates U.S. GAAP requires management to make estimates and assumptions that affect amounts reported in the Condensed Consolidated Financial Statements and accompanying disclosures. Although these estimates are based on management’s best knowledge of current events and actions that the Company may undertake in the future, actual results may be different from those estimates. The Condensed Consolidated Financial Statements reflect all adjustments of a normal, recurring nature that are, in the opinion of management, necessary for a fair presentation of results for these interim periods. Significant Accounting Policies There have been no changes to the Company’s significant accounting policies during the three months ended March 31, 2026, as compared to the significant accounting policies disclosed in Note 1 – Business Overview and Significant Accounting Policies to the Company’s Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025. Recent Accounting Pronouncements There have been no new accounting pronouncements adopted by the Company or new accounting pronouncements issued by the Financial Accounting Standards Board (FASB) during the three months ended March 31, 2026, as compared to the recent accounting pronouncements described in Note 1 to the Company’s Consolidated Financial Statements of the Company’s Annual Report on Form 10-K for the year ended December 31, 2025, that the Company believes are of significance or potential significance to the Company. (2) FINANCIAL INSTRUMENTS All marketable securities were classified as available-for-sale as of March 31, 2026 and December 31, 2025. The following tables show the Company’s cash, cash equivalents, restricted cash equivalents and available-for-sale securities by significant investment category as of the dates presented: 7 Table of Contents BIOMARIN PHARMACEUTICAL INC. NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS (UNAUDITED) - (continued) (In thousands of U.S. Dollars, except per share amounts or as otherwise disclosed) March 31, 2026 Amortized Cost Gross Unrealized Gains Gross Unrealized Losses Aggregate Fair Value Cash and Cash Equivalents Restricted Cash Equivalents Level 1: Cash $ 352,248 $ — $ — $ 352,248 $ 352,248 $ — Level 2: Money market instruments 2,720,187 — — 2,720,187 1,870,187 850,000 Total $ 3,072,435 $ — $ — $ 3,072,435 $ 2,222,435 $ 850,000 December 31, 2025 Amortized Cost Gross Unrealized Gains Gross Unrealized Losses Aggregate Fair Value Cash and Cash Equivalents Short-term Marketable Securities (1) Long-term Marketable Securities (2) Level 1: Cash $ 415,760 $ — $ — $ 415,760 $ 415,760 $ — $ — Level 2: Money market instruments 895,919 — — 895,919 895,919 — — Corporate debt securities 472,572 3,286 ( 3 ) 475,855 — 189,566 286,289 U.S. government agency securities 206,018 1,197 ( 1 ) 207,214 — 59,115 148,099 Asset-backed securities 57,687 420 ( 4 ) 58,103 — 249 57,854 Subtotal 1,632,196 4,903 ( 8 ) 1,637,091 895,919 248,930 492,242 Total $ 2,047,956 $ 4,903 $ ( 8 ) $ 2,052,851 $ 1,311,679 $ 248,930 $ 492,242 (1) The Company’s short-term marketable securities as of December 31, 2025 mature in one year or less, and were liquidated in the first quarter of 2026 to fund the Amicus Therapeutics, Inc. (Amicus) acquisition that closed in April 2026. (2) The Company’s long-term marketable securities as of December 31, 2025 mature between one and five years , and were liquidated in the first quarter of 2026 to fund the Amicus acquisition that closed in April 2026. Restricted Cash Equivalents, Noncurrent Restricted cash equivalents consists of funds held in escrow and restricted in their use to finance a portion of total consideration for the acquisition of Amicus. Refer to Note 13 – Subsequent Events for additional details related to Amicus acquisition. The following table provides a reconciliation of cash, cash equivalents, and restricted cash equivalents reported on the Company’s Condensed Consolidated Balance Sheets that sum to the total of the same such amounts shown in the Condensed Consolidated Statements of Cash Flows: March 31, 2026 Cash and cash equivalents $ 2,222,435 Restricted cash equivalents 850,000 Cash, cash equivalents and restricted cash equivalents $ 3,072,435 8 Table of Contents BIOMARIN PHARMACEUTICAL INC. NOTES TO UNAUDITED CONDENSED CONSOLIDATED FINANCIAL STATEMENTS (In thousands of U.S. Dollars, except per share amounts or as otherwise disclosed) (3) SUPPLEMENTAL FINANCIAL STATEMENTS INFORMATION Inventory consisted of the following: March 31, 2026 December 31, 2025 Raw materials $ 97,850 $ 106,510 Work-in-process 1,054,613 801,061 Finished goods 120,758 391,312 Total inventory $ 1,273,221 $ 1,298,883 Property, Plant and Equipment, Net consisted of the following: March 31, 2026 December 31, 2025 Property, plant and equipment, gross $ 2,052,351 $ 2,026,813 Accumulated depreciation ( 1,094,280 ) ( 1,074,305 ) Total property, plant and equipment, net $ 958,071 $ 952,508 Depreciation expense, net of amounts capitalized into inventory, for the three months ended March 31, 2026 and 2025 was $ 8.8 million and $ 14.1 million, respectively. Intangible Assets, Net consisted of the following: March 31, 2026 December 31, 2025 Finite-lived intangible assets $ 722,360 $ 723,966 Accumulated amortization ( 517,698 ) ( 510,129 ) Net carrying value $ 204,662 $ 213,837 Accounts Payable and Accrued Liabilities consisted of the following: March 31, 2026 December 31, 2025 Accounts payable and accrued operating expenses $ 395,469 $ 312,768 Accrued compensation expense 152,609 219,422 Accrued rebates payable 177,657 166,925 Foreign currency exchange forward contracts 31,679 31,007 Accrued Interest 9,460 907 Lease liability 9,319 8,685 Accrued royalties payable 6,656 7,968 Accrued income taxes 3,010 3,667 Other 7,293 7,682 Total accounts payable and accrued liabilities $ 793,152 $ 759,031 9 Table of Contents BIOMARIN PHARMACEUTICAL INC. NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS (UNAUDITED) - (continued) (In thousands of U.S. Dollars, except per share amounts or as otherwise disclosed) (4) FAIR VALUE MEASUREMENTS The Company measures certain financial assets and liabilities at fair value in accordance with the policy described in Note 1 – Business Overview and Significant Accounting Policies to the Company’s Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025. Other than the Company’s fixed-rate debt disclosed in Note 6 – Debt , there were no financial assets or liabilities that were remeasured using quoted prices in active markets for identical assets (Level 1) as of March 31, 2026 or December 31, 2025. The Company had no financial assets or liabilities that are remeasured on a recurring basis using unobservable inputs that reflect estimates and assumptions (Level 3) as of March 31, 2026 or December 31, 2025. Level 2 assets and liabilities that are remeasured using significant observable inputs consisted of the following, except for derivatives, which are discussed in Note 5 – Derivative Instruments and Hedging Strategies : March 31, 2026 December 31, 2025 Assets: Other current assets: NQDC Plan assets $ 4,055 $ 3,765 Other assets: NQDC Plan assets 40,687 41,689 Total assets $ 44,742 $ 45,454 Liabilities: Accounts payable and accrued liabilities: NQDC Plan liability $ 4,055 $ 3,765 Other long-term liabilities: NQDC Plan liability 40,687 41,689 Total liabilities $ 44,742 $ 45,454 There were no transfers between levels during the three months ended March 31, 2026. (5) DERIVATIVE INSTRUMENTS AND HEDGING STRATEGIES The Company uses foreign currency exchange forward contracts (forward contracts) to protect against the impact of changes in the value of forecasted foreign currency cash flows resulting from revenues and operating expenses denominated in currencies other than the U.S. Dollar (USD), primarily the Euro. Certain of these forward contracts are designated as cash flow hedges and have maturities of up to two years . The Company also enters into forward contracts to manage foreign exchange risk related to asset or liability positions denominated in currencies other than USD. Such forward contracts are considered to be economic hedges, are not designated as hedging instruments and have maturities of up to three months . The Company does not use derivative instruments for speculative trading purposes. The Company is exposed to counterparty credit risk on its derivatives. The Company has established and maintains strict counterparty credit guidelines and enters into hedging agreements with financial institutions that are investment grade or better to minimize the Company’s exposure to potential defaults. The Company is not required to pledge collateral under these agreements. The following table summarizes the aggregate notional amounts for the Company’s derivatives outstanding as of the periods presented. Forward Contracts March 31, 2026 December 31, 2025 Derivatives designated as hedging instruments: Sell $ 1,488,793 $ 1,573,184 Purchase $ 346,573 $ 385,499 Derivatives not designated as hedging instruments: Sell $ 424,423 $ 356,285 Purchase $ 122,441 $ 36,798 10 Table of Contents BIOMARIN PHARMACEUTICAL INC. NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS (UNAUDITED) - (continued) (In thousands of U.S. Dollars, except per share amounts or as otherwise disclosed) The fair value of the Company’s derivatives, which are classified as Level 2 within the fair value hierarchy, were as follows: Balance Sheet Location March 31, 2026 December 31, 2025 Derivatives designated as hedging instruments: Derivative Assets Other current assets $ 25,563 $ 17,585 Other assets 7,665 5,591 Subtotal $ 33,228 $ 23,176 Derivative Liabilities Accounts payable and accrued liabilities $ 30,538 $ 30,134 Other long-term liabilities 6,902 9,905 Subtotal $ 37,440 $ 40,039 Derivatives not designated as hedging instruments: Derivative Assets Other current assets $ 1,830 $ 2,479 Derivative Liabilities Accounts payable and accrued liabilities $ 1,141 $ 873 Total Derivative Assets $ 35,058 $ 25,655 Total Derivative Liabilities $ 38,581 $ 40,912 For additional discussion of fair value measurements, see Note 1 – Business Overview and Significant Accounting Policies to the Company’s Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025. The following tables summarize the impact of gains and losses from the Company's derivatives on its Condensed Consolidated Statements of Comprehensive Income for the periods presented. Three Months Ended March 31, 2026 2025 Derivatives Designated as Cash Flow Hedging Instruments Cash Flow Hedging Gains (Losses) Reclassified into Earnings Net product revenues $ ( 12,412 ) $ 12,545 Operating expenses $ 3,601 $ ( 2,606 ) Derivatives Not Designated as Hedging Instruments Gains (Losses) Recognized in Earnings Operating expenses $ 187 $ ( 12,109 ) As of March 31, 2026, the Company expects to reclassify unrealized losses of $ 5.0 million from Accumulated Other Comprehensive Income (AOCI) to earnings as the forecasted revenues and operating expense transactions occur over the next twelve months. For additional discussion of balances in AOCI see Note 7 – Accumulated Other Comprehensive Income . 11 Table of Contents BIOMARIN PHARMACEUTICAL INC. NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS (UNAUDITED) - (continued) (In thousands of U.S. Dollars, except per share amounts or as otherwise disclosed) (6) DEBT As of March 31, 2026, the Company had outstanding fixed-rate notes for an undiscounted aggregate principal amount of $ 1.5 billion. The notes detailed below incur interest to be paid semi-annually in arrears. The following table summarizes information regarding the Company’s debt: March 31, 2026 December 31, 2025 5.5 % senior unsecured notes due in February 2034 (the 2034 Notes) $ 850,000 $ — Unamortized discount net of deferred offering costs ( 17,407 ) — 2034 Notes, net 832,593 — 1.25 % senior subordinated convertible notes due in May 2027 (the 2027 Notes) $ 600,000 $ 600,000 Unamortized discount net of deferred offering costs ( 2,311 ) ( 2,824 ) 2027 Notes, net 597,689 597,176 Long-term debt, net $ 1,430,282 $ 597,176 Fair value of fixed-rate debt (1) : 2034 Notes $ 839,239 $ — 2027 Notes 578,823 576,267 Total fair value of fixed-rate debt $ 1,418,062 $ 576,267 (1) The fair value of the Company’s fixed-rate long-term debt is based on open-market trades and classified as Level 1 in the fair value hierarchy. For additional discussion of fair value measurements, see Note 1 – Business Overview and Significant Accounting Policies to the Company’s Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025. Interest expense on the Company’s long-term debt consisted of the following: Three Months Ended March 31, 2026 2025 Coupon interest expense $ 8,805 $ 1,875 Accretion of discount on convertible notes 487 485 Amortization of debt issuance costs 279 27 Total interest expense on debt $ 9,571 $ 2,387 2034 Notes In February 2026, the Company issued $ 850.0 million in aggregate principal amount of 5.5 % senior unsecured notes with a maturity date of February 15, 2034, and the proceeds from the issuance were deposited into an escrow account. The 2034 Notes bear interest at the rate of 5.5 % per annum. Interest is payable semi-annually in cash in arrears on February 15 and August 15 of each year, beginning August 15, 2026. Net proceeds from the offering were $ 832.3 million. The Company incurred $ 17.7 million of issuance costs, which were deferred and will be amortized over the life of the 2034 Notes and recorded as interest expense. The indenture governing the Notes contains customary covenants that, among other things, restrict, with certain exceptions, the ability of the Company and its subsidiaries to incur additional debt, pay dividends, make certain other restricted payments, incur debt secured by liens, dispose of assets, engage in consolidations and mergers or sell or transfer all or substantially all of its assets. The offer and sale of the 2034 Notes have not been registered under the Securities Act or any state securities laws and were offered only to qualified institutional buyers as defined in Rule 144A under the Securities Act. 12 Table of Contents BIOMARIN PHARMACEUTICAL INC. NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS (UNAUDITED) - (continued) (In thousands of U.S. Dollars, except per share amounts or as otherwise disclosed) See Note 10 - Debt to the Company’s Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025 for additional information related to the Company’s 2027 Notes and existing credit facility entered into in August 2024. Bridge Commitment In December 2025, the Company entered into a debt financing commitment letter and related fee letter with certain lenders, pursuant to which the lenders committed to provide the Company with debt financing up to approximately $ 3.7 billion (the Bridge Commitment) in the form of a 364-day senior secured bridge loan facility (Bridge Facility), the proceeds of which would be available for the acquisition of Amicus. In connection with the issuance of the 2034 Notes, the Bridge Commitment was reduced to $ 2.8 billion. As a result, the Company recognized approximately $ 5.3 million of commitment fees during the three months ended March 31, 2026 and that is presented as Interest expense on the Condensed Consolidated Statements of Comprehensive Income. As of March 31, 2026, approximately $ 17.5 million in commitment fees were deferred and included in Other Current Assets on the Condensed Consolidated Balance Sheets. Refer to Note 13 – Subsequent Events for additional details related to the Amicus acquisition. (7) ACCUMULATED OTHER COMPREHENSIVE INCOME The following tables summarize changes in the accumulated balances for each component of AOCI, including current-period other comprehensive income and reclassifications out of AOCI, for the periods presented. Three Months Ended March 31, 2026 Unrealized Gains (Losses) on Cash Flow Hedges Unrealized Gains (Losses) on Available-for-Sale Debt Securities Total AOCI balance as of December 31, 2025 $ ( 17,244 ) $ 3,771 $ ( 13,473 ) Other comprehensive income (loss) before reclassifications 4,196 ( 4,895 ) ( 699 ) Less: gain (loss) reclassified from AOCI ( 8,811 ) — ( 8,811 ) Tax effect — 1,124 1,124 Net current-period other comprehensive income (loss) 13,007 ( 3,771 ) 9,236 AOCI balance as of March 31, 2026 $ ( 4,237 ) $ — $ ( 4,237 ) Three Months Ended March 31, 2025 Unrealized Gains (Losses) on Cash Flow Hedges Unrealized Gains (Losses) on Available-for-Sale Debt Securities Total AOCI balance as of December 31, 2024 $ 59,824 $ 1,829 $ 61,653 Other comprehensive income (loss) before reclassifications ( 33,652 ) 1,434 ( 32,218 ) Less: gain (loss) reclassified from AOCI 9,939 — 9,939 Tax effect — ( 345 ) ( 345 ) Net current-period other comprehensive income (loss) ( 43,591 ) 1,089 ( 42,502 ) AOCI balance as of March 31, 2025 $ 16,233 $ 2,918 $ 19,151 For additional discussion of reclassifications from AOCI see Note 5 – Derivative Instruments and Hedging Strategies . 13 Table of Contents BIOMARIN PHARMACEUTICAL INC. NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS (UNAUDITED) - (continued) (In thousands of U.S. Dollars, except per share amounts or as otherwise disclosed) (8) SEGMENT INFORMATION The Company operates and is managed as one business segment which derives revenue from activities related to the development and commercialization of innovative therapies for people with serious and life-threatening rare diseases and medical conditions. The Company’s commercial organization is responsible for marketing its approved products worldwide. The Company’s research and development (R&D) organization is responsible for research and discovery of new product candidates and supporting the development and registration efforts for potential new products. The Company’s technical operations group is responsible for the development of manufacturing processes, supplying clinical drug product, and the manufacturing and distribution of the Company’s commercial products. The Company is also supported by corporate staff functions. The Company’s Chief Executive Officer, as the Chief Operating Decision Maker (CODM), manages and allocates resources to the operations of the total company by assessing the overall level of resources available and how to best allocate them to support the Company’s long-term company-wide strategic goals. In making this decision, the CODM uses consolidated financial information for the purposes of evaluating performance, allocating resources, setting incentive compensation targets and planning and forecasting for future periods. The key measure of segment profit or loss used by the CODM to allocate resources and assess the Company's performance is its Consolidated Net Income, as reported on the Condensed Consolidated Statements of Comprehensive Income. The CODM's analysis includes a comparison to budgeted results. Segment assets provided to the CODM are consistent with those reported on the Condensed Consolidated Balance Sheets with particular emphasis on the Company's available liquidity including cash, cash equivalents, investments, accounts receivable and inventory. The following table includes information about segment revenue, significant segment expenses, and segment measure of profitability: Three Months Ended March 31, 2026 2025 Total revenues $ 766,208 $ 745,145 Less: Cost of sales 194,999 151,558 R&D expenses Research and early pipeline 87,291 90,470 Later-stage clinical programs 36,177 14,291 Marketed products 55,328 53,970 SG&A expenses S&M expenses 124,927 102,531 G&A expenses 133,363 103,585 Other segment expense, net (1) 28,596 43,054 Net income $ 105,527 $ 185,686 (1) Other segment expense, net during the three months ended March 31, 2026 and 2025 include intangible asset amortization, interest income and expense, other income (expense) and income tax expense. 14 Table of Contents BIOMARIN PHARMACEUTICAL INC. NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS (UNAUDITED) - (continued) (In thousands of U.S. Dollars, except per share amounts or as otherwise disclosed) The following table presents Total Revenues and disaggregates Net Product Revenues by product. Three Months Ended March 31, 2026 2025 VOXZOGO $ 219,880 $ 213,754 Enzyme Therapies: VIMIZIM 210,204 188,346 NAGLAZYME 130,074 114,290 PALYNZIQ 89,558 93,269 BRINEURA 47,104 40,362 ALDURAZYME 36,742 48,977 KUVAN 23,880 25,136 ROCTAVIAN 2,636 10,510 Total net product revenues 760,078 734,644 Royalty and other revenues 6,130 10,501 Total revenues $ 766,208 $ 745,145 The Company considers there to be revenue concentration risks for regions where Net Product Revenues exceed 10% of consolidated Net Product Revenues. The concentration of the Company’s Net Product Revenues within the regions below may have a material adverse effect on the Company’s revenues and results of operations if sales in the respective regions experience difficulties. The table below disaggregates total Net Product Revenues by geographic region, which is based on patient location for the Company's commercial products sold directly by the Company, except for ALDURAZYME, which is distributed, marketed and sold exclusively by Sanofi worldwide. Three Months Ended March 31, 2026 2025 United States $ 242,944 $ 241,694 Europe 218,836 229,578 Latin America 108,164 88,282 Rest of world 153,392 126,113 Total net product revenues marketed by the Company 723,336 685,667 ALDURAZYME net product revenues marketed by Sanofi 36,742 48,977 Total net product revenues $ 760,078 $ 734,644 The following table illustrates the percentage of the Company’s total Net Product Revenues attributed to the Company’s largest customers for the periods presented. Three Months Ended March 31, 2026 2025 Customer A 15 % 14 % Customer B 11 12 Customer C 10 11 Total 36 % 37 % 15 Table of Contents BIOMARIN PHARMACEUTICAL INC. NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS (UNAUDITED) - (continued) (In thousands of U.S. Dollars, except per share amounts or as otherwise disclosed) Concentration Information On a consolidated basis, one customer accounted for 20 % of the Company’s March 31, 2026 accounts receivable balance compared to December 31, 2025, when two customers accounted for 20 % and 10 % of the accounts receivable balance, respectively. As of March 31, 2026, and December 31, 2025, the accounts receivable balance for Sanofi included $ 152.6 million and $ 148.0 million, respectively, of unbilled accounts receivable, which becomes payable to the Company when the product is sold through by Sanofi. The Company does not require collateral from its customers, but does perform periodic credit evaluations of its customers’ financial condition and requires prepayments in certain circumstances. The Company is mindful that conditions in the current macroeconomic environment, such as inflation, changes in interest and foreign currency exchange rates, natural disasters, geopolitical instability, wars and military conflicts, impact of new or increased tariffs and escalating trade tensions, regulatory uncertainty, and supply chain disruptions, could affect the Company’s ability to achieve its goals. In addition, the Company sells its products in countries that face economic volatility and weakness. Although the Company has historically collected receivables from customers in such countries, sustained weakness or further deterioration of the local economies and currencies may cause customers in those countries to delay payment or be unable to pay for the Company’s products. The Company believes that the allowances for doubtful accounts related to these countries, if any, are adequate as of March 31, 2026 based on its analysis of the specific business circumstances and expectations of collection for each of the underlying accounts in these countries. The Company will continue to monitor these conditions and will attempt to adjust its business processes, as appropriate, to mitigate macroeconomic risks to its business. (9) STOCK-BASED COMPENSATION The Company has stockholder-approved equity incentive plans that provide for the granting of restricted stock units (RSUs) and stock options as well as other forms of equity compensation to its employees, officers and non-employee directors. The Company also has an Employee Stock Purchase Plan (ESPP). Compensation expense included in the Company’s Condensed Consolidated Statements of Comprehensive Income for all stock-based compensation arrangements was as follows: Three Months Ended March 31, 2026 2025 Cost of sales $ 3,592 $ 2,191 Research and development 11,654 12,127 Selling, general and administrative 28,212 23,382 Total stock-based compensation expense $ 43,458 $ 37,700 (10) RESTRUCTURING In connection with the Company’s plan to simplify its organizational design and strategic initiatives, the Company committed to plans in the first quarter of 2026 to reduce its global workforce. These reductions are expected to be substantially complete by the end of 2026. The reorganization plan and strategic initiatives comprised of severance charges of $ 8.3 million that were included in Selling, General and Administrative in the Condensed Consolidated Financial Statements of Comprehensive Income during the three months ended March 31, 2026. The following unpaid balance as of March 31, 2026 was recorde d to Accounts Payable and Accrued Liabilities on the Condensed Consolidated Balance Sheets: Severance and related costs Balance as of December 31, 2025 $ 3,180 Severance charges 8,312 Payments ( 205 ) Balance as of March 31, 2026 $ 11,287 (11) EARNINGS PER COMMON SHARE Potentially issuable shares of common stock include shares issuable upon the exercise of outstanding employee stock option awards, common stock issuable under the ESPP, unvested RSUs and contingent issuances of common stock related to the Company’s convertible debt. 16 Table of Contents BIOMARIN PHARMACEUTICAL INC. NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS (UNAUDITED) - (continued) (In thousands of U.S. Dollars, except per share amounts or as otherwise disclosed) The following table sets forth the computation of basic and diluted earnings per common share (common shares in thousands): Three Months Ended March 31, 2026 2025 Numerator: Net income, basic $ 105,527 $ 185,686 Add: Interest expense, net of tax, on the Company's convertible debt 1,834 1,833 Net income, diluted $ 107,361 $ 187,519 Denominator: Weighted-average common shares outstanding, basic 192,497 190,967 Effect of dilutive securities: Common stock issuable under the Company's equity incentive plans 809 1,142 Common stock issuable under the Company’s convertible debt 4,365 4,365 Weighted-average common shares outstanding, diluted 197,671 196,474 Earnings per common share, basic $ 0.55 $ 0.97 Earnings per common share, diluted $ 0.54 $ 0.95 In addition to the equity instruments included in the table above, the table below presents potential shares of common stock that were excluded from the computation of diluted earnings per common share as they were anti-dilutive (in thousands): Three Months Ended March 31, 2026 2025 Common stock issuable under the Company's equity incentive plans 13,803 11,686 (12) COMMITMENTS AND CONTINGENCIES Contingencies From time to time the Company is involved in legal actions arising in the normal course of its business. The process of resolving matters through litigation or other means is inherently uncertain and it is possible that an unfavorable resolution of these matters could adversely affect the Company, its results of operations, financial condition or cash flows. The Company’s general practice is to expense legal fees as services are rendered in connection with legal matters, and to accrue for liabilities when losses are probable and reasonably estimable based on existing information. The Company accrues for the best estimate of a loss within a range; however, if no estimate in the range is better than any other, then the minimum amount in the range is accrued. Liabilities are evaluated and refined each reporting period as additional information is known. Any receivables for insurance recoveries for these liability claims are recorded as assets when it is probable that a recovery will be realized. As first disclosed in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023, the Company received a subpoena from the U.S. Department of Justice (DOJ) requesting that the Company produce certain documents regarding sponsored testing programs relating to VIMIZIM and NAGLAZYME. The Company has produced the requested documents in response to the subpoena and is cooperating fully. The Company is unable to make any assurances regarding the outcome of the investigation by the DOJ, or the impact, if any, that such investigation may have on the Company’s business and financial statements. Other Commitments The Company uses experts and laboratories at universities and other institutions to perform certain R&D activities. These amounts are recorded as R&D expense as services are provided. In the normal course of business, the Company enters into various firm purchase commitments primarily to procure active pharmaceutical ingredients, certain inventory-related items and certain third-party R&D services, production services and facility construction services. As of March 31, 2026, such commitments were estimated at $ 593.6 million, of which $ 327.8 million is expected to be paid in 2026 as underlying goods and services are received. The Company has also licensed technology from third parties, for which it is required to pay royalties upon future sales, subject to certain annual minimums. 17 Table of Contents BIOMARIN PHARMACEUTICAL INC. NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS (UNAUDITED) - (continued) (In thousands of U.S. Dollars, except per share amounts or as otherwise disclosed) (13) SUBSEQUENT EVENTS On April 27, 2026, the Company completed its previously announced acquisition of Amicus, a publicly traded, global, biotechnology company for $ 14.50 per share in an all cash transaction for a total equity value of approximately $ 4.8 billion. In connection with the acquisition, the Company also repaid in full all outstanding loans, together with interest and all other amounts due in connection with such repayment, of Amicus for approximately $ 433.0 million. The acquisition is expected to strengthen the Company's commercial portfolio by adding two new treatments to its existing portfolio of medicines that target lysosomal storage diseases: GALAFOLD (migalastat), the first oral treatment for Fabry disease, and POMBILITI (cipaglucosidase alfa-atga) + OPFOLDA (miglustat), a two-component therapy for Pompe disease. In connection with the acquisition, the Company also received U.S. rights to DMX-200, a potential first-in-class investigational small molecule for the treatment of focal segmental glomerulosclerosis (FSGS), a rare fatal kidney disease in Phase 3 development. The transaction was financed through a combination of cash on hand and non-convertible debt financing. In connection with the acquisition, the Company obtained senior secured term loan facilities for $ 2.8 billion in aggregate principal and a new $ 600.0 million senior secured revolving credit facility. Borrowings under the term loan facilities and the 2026 revolving facility bear interest at the applicable interest rates specified in the governing credit agreement (the 2026 Credit Agreement). The 2026 Credit Agreement contains customary affirmative and negative covenants, including, among others, covenants that restrict the Company’s ability to incur additional indebtedness, create liens, make investments, pay dividends or make other restricted payments, dispose of assets, and enter into transactions with affiliates, in each case subject to exceptions set forth in the 2026 Credit Agreement. Upon entry into the senior secured term loan facility, the remaining Bridge Commitment was reduced from $ 2.8 billion to zero , and the Company recognized approximately $ 17.5 million of commitment fees that were deferred on the Condensed Consolidated Balance Sheets as of March 31, 2026. The accounting impact of this acquisition and the results of operations for Amicus will be included in the Company’s Consolidated Financial Statements beginning in the second quarter of 2026. The initial accounting for this acquisition is incomplete, pending identification and measurement of the assets acquired and liabilities assumed. 18 Table of Contents Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations The following discussion of our financial condition and results of operations should be read in conjunction with our Condensed Consolidated Financial Statements and the related Notes thereto included in this Quarterly Report on Form 10-Q. This discussion contains forward-looking statements that involve risks and uncertainties. When reviewing the discussion below, you should keep in mind the substantial risks and uncertainties that could impact our business. In particular, we encourage you to review the risks and uncertainties described in “Risk Factors” in Part II, Item 1A of this Quarterly Report on Form 10-Q. These risks and uncertainties could cause actual results to differ significantly from those projected in forward-looking statements contained in this report or implied by past results and trends. Forward-looking statements are statements that attempt to forecast or anticipate future developments in our business, financial condition or results of operations. See the section titled “Forward-Looking Statements” that appears at the beginning of this Quarterly Report on Form 10-Q. These statements, like all statements in this report, speak only as of the date of this Quarterly Report on Form 10-Q (unless another date is indicated), and, except as required by law, we undertake no obligation to update or revise these statements in light of future developments. Our Condensed Consolidated Financial Statements have been prepared in accordance with United States (U.S.) generally accepted accounting principles (U.S. GAAP) and are presented in U.S. Dollars (USD). 19 Table of Contents Management’s Discussion and Analysis of Financial Condition and Results of Operations (continued) (In millions, except as otherwise disclosed) Overview We are a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Our San Rafael, California-based company, founded in 1997, has a proven track record of innovation, with a portfolio of commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, we seek to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. A summary of our commercial products, as of March 31, 2026, is provided below: Commercial Products Indication VOXZOGO (vosoritide) Achondroplasia Enzyme Therapies: VIMIZIM (elosulfase alpha) Mucopolysaccharidosis (MPS) IVA NAGLAZYME (galsulfase) MPS VI PALYNZIQ (pegvaliase-pqpz) Phenylketonuria (PKU) BRINEURA (cerliponase alfa) Neuronal ceroid lipofuscinosis type 2 (CLN2) ALDURAZYME (laronidase) MPS I KUVAN (sapropterin dihydrochloride) PKU ROCTAVIAN (valoctocogene roxaparvovec) (1) Severe Hemophilia A (1) In 2026, we announced that we will no longer market ROCTAVIAN. For additional information related to ROCTAVIAN, see Note 19 to the Consolidated Financial Statements accompanying our Annual Report on Form 10-K for the year ended December 31, 2025. 20 Table of Contents Management’s Discussion and Analysis of Financial Condition and Results of Operations (continued) (In millions, except as otherwise disclosed) Financial Highlights Key components of our results of operations include the following: Three Months Ended March 31, 2026 2025 Total revenues $ 766.2 $ 745.1 Cost of sales $ 195.0 $ 151.6 Research and development (R&D) $ 178.8 $ 158.7 Selling, general and administrative (SG&A) $ 258.3 $ 206.1 Provision for income taxes $ 35.7 $ 52.4 Net income $ 105.5 $ 185.7 See “ Results of Operations ” below for discussion of our results for the periods presented. Uncertainty Relating to Macroeconomic Environment Conditions in the current macroeconomic environment, such as inflation, changes in interest and foreign currency exchange rates, natural disasters, geopolitical instability, wars and military conflicts, impact of new or increased tariffs and escalating trade tensions, regulatory uncertainty, and supply chain disruptions, could impact our global revenue sources and our overall business operations. The extent and duration of such effects remain uncertain and difficult to predict. We are actively monitoring and managing our response and assessing actual and potential impacts to our operating results and financial condition, as well as developments in our business, which could further impact the developments, trends and expectations described below. See the risk factor, “Our business is affected by macroeconomic conditions.” described in “ Risk Factors ” in Part II, Item 1A of this Quarterly Report on Form 10-Q. Recent Developments We continued to grow our commercial business and advance our product candidate pipeline during 2026. We believe that the combination of our internal research programs, partnerships and acquisitions of external assets will allow us to continue to develop and commercialize innovative therapies for patients with serious and life-threatening rare diseases and medical conditions. We periodically conduct strategic portfolio assessment of research and development programs to determine which we believe have the strongest combination of scientific merit, opportunity for commercial success and potential value creation for stockholders. Based on such strategic portfolio assessments, certain programs that do not meet its threshold for further development and commercialization could be discontinued. • In April 2026, we completed the acquisition of Amicus Therapeutics, Inc. (Amicus), a publicly traded, global, biotechnology company for $14.50 per share in an all-cash transaction for a total equity value of approximately $4.8 billion. In connection with the acquisition, the Company also repaid in full all outstanding loans, together with interest and all other amounts due in connection with such repayment, of Amicus for approximately $433.0 million. The acquisition was financed through a combination of cash on hand and non-convertible debt financing. The acquisition is expected to strengthen our commercial portfolio by adding two new treatments to our existing portfolio of medicines that target lysosomal storage diseases: GALAFOLD (migalastat), the first oral treatment for Fabry disease, and POMBILITI (cipaglucosidase alfa-atga) + OPFOLDA (miglustat), a two-component therapy for Pompe disease. In connection with the acquisition, we also now have U.S. rights to DMX-200, a potential first-in-class investigational small molecule for the treatment of focal segmental glomerulosclerosis (FSGS), a rare fatal kidney disease in Phase 3 development. The accounting impact of this acquisition and the results of operations for Amicus will be included in our Consolidated Financial Statements beginning in the second quarter of 2026. The initial accounting for this acquisition is incomplete, pending identification and measurement of the assets acquired and liabilities assumed. See Note 6 and Note 13 to our accompanying Condensed Consolidated Financial Statements for additional information regarding the acquisition. • In April 2026, in connection with the Amicus acquisition, we obtained senior secured term loan facilities for $2.8 billion in aggregate principal and a new $600.0 million senior secured revolving credit facility. Upon entry into the senior secured term loan facilities, the remaining bridge commitment was reduced from $2.8 billion to zero. In February 2026, we issued $850.0 million in aggregate principal amount of 5.5% senior unsecured notes due 2034 (the 2034 Notes), and the proceeds from the issuance were deposited into an escrow account as of March 31, 2026. The proceeds from the 2034 Notes and the secured term loan facilities were used to finance a portion of the Amicus acquisition that closed in April 2026. No amounts have been drawn under the senior secured revolving credit facility. See “Financial Condition, Liquidity and Capital Resources” below for additional information. 21 Table of Contents Management’s Discussion and Analysis of Financial Condition and Results of Operations (continued) (In millions, except as otherwise disclosed) • In April 2026, the first patient was enrolled in the registration-enabling Phase 2/3 study of BMN 333, our long-acting C-type natriuretic peptide (CNP) for achondroplasia. • In April 2026, we submitted its U.S. supplemental new drug application (sNDA) for full approval of VOXZOGO for achondroplasia. • In March 2026, we presented initial Phase 1/2 data for BMN 351 at the Muscular Dystrophy Association (MDA) Clinical & Scientific Congress demonstrating dose-dependent increases in dystrophin expression at Week 25 biopsy in both the 6 and 9 mg/kg dose cohorts. Clinical biomarkers, including decreases in creatine kinase, suggested improvements in overall muscle health beyond the Week 25 time point, and longer-term outcomes from both NSAA and 6MWT indicated a prevention of functional decline when compared to historical matched controls. • In March 2026, we announced our decision to discontinue dosing and enrollment in our Phase 2 trials for VOXZOGO in Turner Syndrome, SHOX-deficiency and Aggrecan (ACAN)-deficiency. • In February 2026, U.S. Food and Drug Administration (FDA) approved PALYNZIQ for adolescents 12 years of age and older with phenylketonuria (PKU). See the risk factors described under “Business and Operational Risks” section in “Risk Factors” in Part II, Item 1A of this Quarterly Report on Form 10-Q. Results of Operations Net Product Revenues Net Product Revenues consisted of the following: Three Months Ended March 31, 2026 2025 Change VOXZOGO $ 219.9 $ 213.7 $ 6.2 Enzyme Therapies: VIMIZIM 210.2 188.3 21.9 NAGLAZYME 130.1 114.3 15.8 PALYNZIQ 89.6 93.3 (3.7) BRINEURA 47.1 40.4 6.7 ALDURAZYME 36.7 49.0 (12.3) KUVAN 23.9 25.1 (1.2) ROCTAVIAN 2.6 10.5 (7.9) Total net product revenues $ 760.1 $ 734.6 $ 25.5 Net Product Revenues include revenues generated from our commercial products. In the U.S., our commercial products, except for PALYNZIQ and ALDURAZYME, are generally sold to specialty pharmacies or end users, such as hospitals, which act as retailers. PALYNZIQ is distributed in the U.S. through certain certified specialty pharmacies under the PALYNZIQ Risk Evaluation and Mitigation Strategy program, and ALDURAZYME is marketed worldwide by Sanofi. Outside the U.S., our commercial products are sold to authorized distributors or directly to government purchasers or hospitals, which act as the end users. The increase in Net Product Revenues for the three months ended March 31, 2026 as compared to the three months ended March 31, 2025 was primarily attributed to the following: • VIMIZIM and NAGLAZYME: higher sales volume due to timing of orders in countries that place large government orders, primarily from countries in the Middle East and Latin America; 22 Table of Contents Management’s Discussion and Analysis of Financial Condition and Results of Operations (continued) (In millions of U.S. dollars, except as otherwise disclosed) • BRINEURA: higher sales volume due to new patients initiating therapy across all regions and timing of orders in countries that place large government orders; and • VOXZOGO: higher sales volume from new patients initiating therapy across all regions. These increases were partially offset by the following: • ALDURAZYME: lower sales volume due to timing of order fulfillment to Sanofi; and • ROCTAVIAN: lower sales volume due to our voluntary withdrawal of the product from the market announced in the first quarter of 2026. In certain countries, governments place large periodic orders for our products. We expect that the timing of these large government orders will continue to be inconsistent, which has created in the past and may continue to create significant period to period variation in our revenues. We expect Total Revenues to increase over the next 12 months following the Amicus acquisition that closed in April 2026. With respect to VOXZOGO, see also the risk factors “Our success depends on our ability to manage our growth and execute our corporate strategy.” and “If we fail to compete successfully with respect to product sales, we may be unable to generate sufficient sales to recover our expenses related to the development of a product program or to justify continued marketing of a product and our revenues could be adversely affected.” in "Risk Factors" in Part II, Item 1A of this Quarterly Report for additional information on risk factors that could impact our business and operations. We face exposure to movements in foreign currency exchange rates, and use foreign currency exchange forward contracts to hedge a percentage of our foreign currency exposure, primarily the Euro. Certain currencies are not included in our hedging program, such as the Argentine Peso. With respect to the risks posed by fluctuations of both hedged and unhedged currencies against the U.S. dollar (USD), see the risk factor “Our international operations pose currency risks, which may adversely affect our operating results and net income” in “Risk Factors” included in Part II, Item 1A of this Quarterly Report for additional information. The following table shows our Net Product Revenues denominated in USD and foreign currencies: Three Months Ended March 31, 2026 2025 Change Sales denominated in USD $ 362.7 $ 358.3 $ 4.4 Sales denominated in foreign currencies 397.4 376.3 21.1 Total net product revenues $ 760.1 $ 734.6 $ 25.5 Three Months Ended March 31, 2026 2025 Change Favorable (unfavorable) impact of foreign currency exchange rates on product sales denominated in currencies other than USD $ 8.6 $ (13.6) $ 22.2 The favorable impact for the three months ended March 31, 2026 was primarily driven by strengthening of the Euro and Mexican Peso, partially offset by weakening of the Argentine Peso and the Japanese Yen. The unfavorable impact for the three months ended March 31, 2025 was primarily driven by weakening of the Brazilian Real, Euro, Colombian Peso and Argentine Peso. Cost of Sales and Gross Margin Cost of Sales includes raw materials, personnel and facility and other costs associated with manufacturing our commercial products. These costs include production materials, production costs at our manufacturing facilities, third-party manufacturing costs, amortization of technology transfer intangible assets and internal and external final formulation and packaging costs. Cost of Sales also includes royalties payable to third parties based on sales of our products, idle plant costs and charges for inventory write downs. 23 Table of Contents Management’s Discussion and Analysis of Financial Condition and Results of Operations (continued) (In millions of U.S. dollars, except as otherwise disclosed) The following table summarizes our Cost of Sales and Gross Margin: Three Months Ended March 31, 2026 2025 Change Total revenues $ 766.2 $ 745.1 $ 21.1 Cost of sales $ 195.0 $ 151.6 $ 43.4 Gross margin 74.5 % 79.7 % (5.2) % Cost of Sales increased and Gross Margin decreased in the three months ended March 31, 2026 as compared to the three months ended March 31, 2025, primarily due to a $31.0 million charge associated with an unsuccessful process qualification campaign to expand NAGLAZYME manufacturing capabilities. Research and Development R&D expense includes costs associated with the research and development of product candidates and post-marketing research commitments related to our commercial products. R&D expense primarily includes preclinical and clinical studies, personnel and raw materials costs associated with manufacturing clinical product, quality control and assurance, other R&D activities, R&D facilities and regulatory costs. We group all of our R&D activities and related expense into three categories: (i) Research and early pipeline, (ii) Later-stage clinical programs and (iii) Marketed products as follows: Category Description Research and early pipeline R&D expense incurred in activities substantially in support of early research through the completion of phase 2 clinical trials, including drug discovery, toxicology, pharmacokinetics and drug metabolism and process development. Later-stage clinical programs R&D expense incurred in or related to phase 3 clinical programs intended to result in registration of a new product or a new indication for an existing product primarily in the U.S. or the EU. Marketed products R&D expense incurred in support of our marketed products that are authorized to be sold primarily in the U.S. or the EU. Includes clinical trials designed to gather information on product safety (certain of which may be required by regulatory authorities) and their product characteristics after regulatory approval has been obtained, as well as the costs of obtaining regulatory approval of a product in a new market after approval in either the U.S. or EU has been obtained. We manage our R&D expense by identifying the R&D activities we anticipate will be performed during a given period and then prioritizing efforts based on scientific data, probability of successful development, market potential, available human and capital resources and other similar considerations. We continually review our product pipeline and the development status of product candidates and, as necessary, reallocate resources among the research and development portfolio that we believe will best support the future growth of our business. We continuously evaluate the recoverability of costs associated with pre-launch or pre-qualification manufacturing activities, if any, and capitalize the costs incurred related to those activities if we determine that recoverability is probable and therefore future revenues are expected. If the related product candidate's marketing application is rejected by the applicable regulators and the likelihood of future revenues for a product candidate become uncertain, the related manufacturing costs are expensed as R&D expenses. 24 Table of Contents Management’s Discussion and Analysis of Financial Condition and Results of Operations (continued) (In millions of U.S. dollars, except as otherwise disclosed) R&D expense consisted of the following: Three Months Ended March 31, 2026 2025 Change Research and early pipeline $ 87.3 $ 90.4 $ (3.1) Later-stage clinical programs 36.2 14.3 21.9 Marketed products 55.3 54.0 1.3 Total R&D expense $ 178.8 $ 158.7 $ 20.1 The increase in R&D expense for the three months ended March 31, 2026 as compared to the three months ended March 31, 2025 was primarily due to higher spend on BMN 401, a later-stage clinical program acquired in the third quarter of 2025. Selling, General and Administrative Sales and marketing (S&M) expense primarily consisted of employee-related expenses for our sales group, brand marketing, patient support groups and pre-commercialization expenses related to our product candidates. General and administrative (G&A) expense primarily consisted of corporate support and other administrative expenses, including employee-related expenses. SG&A expense consisted of the following: Three Months Ended March 31, 2026 2025 Change S&M $ 124.9 $ 102.5 $ 22.4 G&A 133.4 103.6 29.8 Total SG&A expense $ 258.3 $ 206.1 $ 52.2 S&M expense consisted of the following: Three Months Ended March 31, 2026 2025 Change Enzyme Therapies $ 63.3 $ 48.6 $ 14.7 VOXZOGO 44.3 34.5 9.8 Other 17.3 19.4 (2.1) Total S&M expense $ 124.9 $ 102.5 $ 22.4 The increase in S&M expense for the three months ended March 31, 2026 as compared to the three months ended March 31, 2025 was primarily due to increased spending related to global expansion of Enzyme Therapies and VOXZOGO. The increase in G&A expense for the three months ended March 31, 2026 as compared to the three months ended March 31, 2025 was primarily due to incremental administrative costs related to ongoing support of corporate initiatives and pre-close costs related to Amicus acquisition. Intangible Asset Amortization Intangible Asset Amortization was as follows: Three Months Ended March 31, 2026 2025 Change Amortization of intangible assets $ 4.5 $ 4.8 $ (0.3) 25 Table of Contents Management’s Discussion and Analysis of Financial Condition and Results of Operations (continued) (In millions of U.S. dollars, except as otherwise disclosed) Amortization of Intangible Assets for the three months ended March 31, 2026 as compared to the three months ended March 31, 2025 was relatively flat. We expect Intangible Asset Amortization to significantly increase over the next 12 months following the Amicus acquisition that closed in April 2026. Interest Income We invest our cash equivalents and investments in U.S. government securities and other high credit quality debt securities in order to limit default and market risk. Three Months Ended March 31, 2026 2025 Change Interest income $ 22.6 $ 19.0 $ 3.6 The increase in Interest Income for the three months ended March 31, 2026 as compared to the three months ended March 31, 2025 was mainly due to higher levels of cash, cash equivalents and investment balances. The $850.0 million proceeds from the issuance of 2034 Notes in the first quarter of 2026 were held in escrow and invested in money market funds as of March 31, 2026. We expect Interest Income to decrease over the next 12 months due to lower cash and investment balances following the Amicus acquisition that closed in April 2026. Interest Expense We incur interest expense primarily on our long-term debt. Interest Expense for the periods presented was as follows: Three Months Ended March 31, 2026 2025 Change Interest expense $ 15.0 $ 2.9 $ 12.1 The increase in Interest Expense for the three months ended March 31, 2026 as compared to the three months ended March 31, 2025 was primarily due to issuance of 2034 Notes for $850.0 million aggregate principal in February 2026, and $5.3 million of bridge commitment fees recognized during the three months ended March 31, 2026. We expect Interest Expense to significantly increase over the next 12 months due to the financing related to the Amicus acquisition that closed in April 2026, including the new term loans issued in April 2026. See Note 6 and Note 13 to our accompanying Condensed Consolidated Financial Statements for additional information regarding our debt. Other Income (Expense), Net Other Income (Expense), Net for the periods presented was as follows: Three Months Ended March 31, 2026 2025 Change Other income (expense), net $ 4.0 $ (2.0) $ 6.0 The increase in Other Income (Expense), Net for the three months ended March 31, 2026 as compared to the three months ended March 31, 2025 was primarily due to gain on sale of marketable securities resulting from the sale of our short-term and long-term investments to fund the Amicus acquisition that closed in April 2026. 26 Table of Contents Management’s Discussion and Analysis of Financial Condition and Results of Operations (continued) (In millions of U.S. dollars, except as otherwise disclosed) Provision for Income Taxes The Provision for Income Taxes for the periods presented was as follows: Three Months Ended March 31, 2026 2025 Change Provision for income taxes $ 35.7 $ 52.4 $ (16.7) The decrease in Provision for Income Taxes for the three months ended March 31, 2026 as compared to the three months ended March 31, 2025 was primarily due to lower pre-tax income, partially offset by higher tax expense related to stock based compensation. Financial Condition, Liquidity and Capital Resources Our cash, cash equivalents, restricted cash equivalents and investments as of March 31, 2026 and December 31, 2025 were as follows: March 31, 2026 December 31, 2025 Change Cash and cash equivalents $ 2,222.4 $ 1,311.7 $ 910.7 Short-term investments — 248.9 (248.9) Long-term investments — 492.2 (492.2) Restricted cash equivalents 850.0 — 850.0 Total cash, cash equivalents, restricted cash equivalents and investments $ 3,072.4 $ 2,052.8 $ 1,019.6 We believe cash generated from sales of our commercial products, in addition to our cash, cash equivalents and restricted cash equivalents and external financings, will be sufficient to satisfy our liquidity requirements for at least the next 12 months, including our debt service commitments relating to the Amicus acquisition. We believe we will meet longer-term expected future cash requirements and obligations through a combination of cash flows from operating activities and available cash. We will need to raise additional funds by issuing equity, debt or convertible securities, taking loans or entering into collaborative or other agreements if we are unable to satisfy our liquidity requirements. For example, we may require additional financing to fund the repayment of our outstanding indebtedness, future milestone payments and our future operations, including the commercialization of our products and product candidates currently under development, preclinical studies and clinical trials, and potential licenses and acquisitions. The timing and mix of our funding alternatives could change depending on many factors, including how much we elect to spend on our development programs, potential licenses and acquisitions of complementary technologies, products and companies or if we settle our long-term debt in cash. In addition, depending on prevailing market conditions, our liquidity requirements, contractual restrictions, and other factors, we may also from time to time seek to retire or purchase our outstanding convertible debt through cash purchases and/or exchanges for equity securities, in open market purchases, privately negotiated transactions or otherwise. We are mindful that conditions in the current macroeconomic environment, such as inflation, changes in interest and foreign currency exchange rates, natural disasters, geopolitical instability, wars and military conflicts, impact of new or increased tariffs and escalating trade tensions, regulatory uncertainty, and supply chain disruptions could affect our ability to achieve our goals. In addition, we sell our products in certain countries that face economic volatility and weakness. Although we have historically collected receivables from customers in such countries, sustained weakness or further deterioration of the local economies and currencies may cause customers in those countries to be unable to pay for our products. We will continue to monitor these conditions and will attempt to adjust our business processes, as appropriate, to mitigate macroeconomic risks to our business. 27 Table of Contents Management’s Discussion and Analysis of Financial Condition and Results of Operations (continued) (In millions of U.S. dollars, except as otherwise disclosed) Our cash flows are summarized as follows: Three Months Ended March 31, 2026 2025 Change Net cash provided by operating activities $ 220.7 $ 174.4 $ 46.3 Net cash provided by (used in) investing activities $ 725.5 $ (28.2) $ 753.7 Net cash provided by (used in) financing activities $ 813.2 $ (38.8) $ 852.0 The increase in net cash provided by operating activities in the three months ended March 31, 2026 compared to March 31, 2025 was primarily attributed to decrease in inventory campaigns and timing of cash receipts from our customers, partially offset by decrease in net income. The increase in net cash provided by investing activities in the three months ended March 31, 2026 compared to March 31, 2025 was primarily attributable to higher net maturities of available-for-sale (AFS) securities. All of the proceeds from the sale of these AFS securities were held as cash and subsequently used to finance a portion of the Amicus acquisition that closed in April 2026. The increase in net cash provided by financing activities in the three months ended March 31, 2026 compared to March 31, 2025 was primarily attributable to proceeds from the issuance of 2034 Notes. The proceeds from the 2034 Notes were held in escrow as of March 31, 2026 and subsequently used to finance a portion of the Amicus acquisition that closed in April 2026. Financing Our $1.5 billion (undiscounted) of long-term debt as of March 31, 2026 will impact our liquidity due to the semi-annual cash interest payments as well as the repayment of the principal amount. As of March 31, 2026, our indebtedness consisted of our 5.5% senior unsecured notes due to be repaid in cash at maturity in February 2034, and senior subordinated convertible notes due in 2027, which, if not converted, will be required to be repaid in cash at maturity in May 2027. In December 2025, we entered into a debt financing commitment letter and related fee letter with certain lenders, pursuant to which the lenders committed to provide the Company with debt financing up to approximately $3.7 billion (the Bridge Commitment) in the form of a 364-day senior secured bridge loan facility (Bridge Facility), the proceeds of which would be available for the acquisition of Amicus. In connection with the issuance of the 2034 Notes in February 2026, the Bridge Commitment was reduced from $3.7 billion to $2.8 billion. In April 2026, in connection with the Amicus acquisition, we incurred $2.0 billion of indebtedness under a senior secured term loan B facility and $800.0 million of indebtedness under a senior secured term loan A facility (together Term Facilities) and entered into a $600.0 million senior secured revolving credit facility (2026 Revolving Facility and, together with the Term Facilities, the 2026 Senior Secured Credit Facilities). Upon entry into the Term Facilities, the remaining Bridge Commitment was reduced to zero. The term loan B facility matures in April 2033, and the term loan A facility and 2026 Revolving Facility each mature in April 2031. Borrowings under the Term Facilities and the 2026 Revolving Facility bear interest at the applicable interest rates specified in the credit agreement governing the 2026 Senior Secured Credit Facilities (the 2026 Credit Agreement). The 2026 Credit Agreement contains customary affirmative and negative covenants, including, among others, covenants that restrict our ability to incur additional indebtedness, create liens, make investments, pay dividends or make other restricted payments, dispose of assets, and enter into transactions with affiliates, in each case subject to exceptions set forth in the 2026 Credit Agreement. In connection with the Amicus acquisition, we used approximately $1.6 billion of cash on hand, together with net proceeds from the 2034 Notes and borrowings under the Term Facilities, to fund the aggregate cash consideration of approximately $5.2 billion. As a result, our total indebtedness increased from approximately $1.5 billion as of March 31, 2026 to approximately $4.3 billion, and our material cash requirements have increased significantly, including semi-annual fixed-rate interest payments on the 2027 Notes and 2034 Notes, variable-rate interest payments on the Term Facilities, and scheduled principal repayments. The $600.0 million 2026 Revolving Facility remains available for working capital and general corporate purposes. In August 2024, we entered into an unsecured revolving credit facility (2024 Revolving Facility) providing for $600.0 million in revolving loan commitments. The 2024 Revolving Facility was intended to finance ongoing working capital needs and for other general corporate purposes. The credit facility contains financial covenants including a maximum total net leverage ratio and a minimum interest coverage ratio. The 2024 Revolving Facility was to mature in August 2029. As of March 31, 2026, there were no amounts outstanding under the 2024 Revolving Facility and we were in compliance with all covenants. In April 2026, in connection with the entry into the 2026 Senior Secured Credit Facilities, we terminated the 2024 Revolving Facility. 28 Table of Contents Management’s Discussion and Analysis of Financial Condition and Results of Operations (continued) (In millions of U.S. dollars, except as otherwise disclosed) For additional information related to our long-term debt, see Note 6 and Note 13 to our accompanying Condensed Consolidated Financial Statements and Note 10 to the Consolidated Financial Statements accompanying our Annual Report on Form 10-K for the year ended December 31, 2025. Material Cash Requirements Purchase and Lease Obligations, and Unrecognized Tax Benefits As of March 31, 2026, we had obligations of approximately $593.6 million, of which $327.8 million is expected to be paid in 2026. Our purchase obligations are primarily related to firm purchase commitments entered into in the normal course of business to procure active pharmaceutical ingredients, certain inventory-related items, certain third-party R&D services, production services and facility construction services. Our lease commitments and unrecognized tax benefits as of March 31, 2026 have not materially changed from those discussed in “Financial Condition, Liquidity and Capital Resources” in Part II, Item 7 of our Annual Report on Form 10-K for the year ended December 31, 2025. See Note 1 2 to our accompanying Condensed Consolidated Financial Statements for additional information on our commitments. Critical Accounting Estimates In preparing our Condensed Consolidated Financial Statements in accordance with U.S. GAAP and pursuant to the rules and regulations promulgated by the Securities and Exchange Commission (the SEC), we make assumptions, judgments and estimates that can have a significant impact on our net income/loss and affect the reported amounts of certain assets, liabilities, revenues and expenses, and related disclosures. On an ongoing basis, we evaluate our estimates and discuss our critical accounting policies and estimates with the Audit Committee of our Board of Directors. We base our estimates on historical experience and various other assumptions that we believe to be reasonable under the circumstances. Actual results could differ materially from these estimates under different assumptions or conditions. Historically, our assumptions, judgments and estimates relative to our critical accounting estimates have not differed materially from actual results. There have been no significant changes to our critical accounting estimates during the three months ended March 31, 2026, compared to those disclosed in “Management’s Discussion and Analysis of Financial Condition and Results of Operations” included in our Annual Report on Form 10-K for the year ended December 31, 2025, filed with the SEC on February 26, 2026. Recent Accounting Pronouncements See Note 1 to our accompanying Condensed Consolidated Financial Statements for a description of recent accounting pronouncements, if any, and our expectation of their impact on our results of operations and financial condition. 29 Table of Contents Item 3. Quantitative and Qualitative Disclosures About Market Risk Our market risks during the three months ended March 31, 2026 have not materially changed from those discussed in Part II, Item 7A of our Annual Report on Form 10-K for the year ended December 31, 2025. Item 4. Controls and Procedures (a) Controls and Procedures An evaluation was carried out, under the supervision of and with the participation of our management, including our Chief Executive Officer and our Chief Financial Officer, of the effectiveness of our disclosure controls and procedures (as defined in Rules 13a-15(e) and 15d-15(e) under the Securities Exchange Act of 1934, as amended (the Exchange Act)), as of the end of the period covered by this report. Based on the evaluation, our Chief Executive Officer and our Chief Financial Officer have concluded that our disclosure controls and procedures were effective, at the reasonable assurance level, as of March 31, 2026. In designing and evaluating our disclosure controls and procedures, our management recognizes that any controls and procedures, no matter how well designed and operated, can provide only reasonable assurance of achieving the desired control objectives, and our management must apply its judgment in evaluating the cost-benefit relationship of possible controls and procedures. Accordingly, our disclosure controls and procedures are designed to provide reasonable, not absolute, assurance that the objectives of our disclosure controls system are met. (b) Changes in Internal Control over Financial Reporting Except as otherwise noted, there were no changes in our internal control over financial reporting, as such term is defined in Rules 13a-15(f) and 15d-15(f) under the Exchange Act, during our most recently completed quarter that have materially affected or are reasonably likely to materially affect our internal control over financial reporting. We continue to utilize the Committee of Sponsoring Organizations of the Treadway Commission (COSO) 2013 Framework on internal control. We rely extensively on information systems and technology to manage our business, including integrated supply chain operations, and global consolidated financial results. In January 2025, we began deploying a new global enterprise resource planning (ERP) system at certain subsidiaries, replacing existing operating and financial systems with implementation scheduled to occur in phases through 2026. The final phase of the deployment was completed in January 2026. The ERP system is designed to accurately maintain our financial records, support integrated supply chain and other operational functionality, and provide timely information to our management team related to the operation of the business. We have updated our internal control over financial reporting, as necessary, to accommodate related changes in our financial management processes resulting from this implementation. As the post-implementation activities take place, we will continue to have changes to certain of our processes and procedures, and we will evaluate quarterly whether the changes materially affect our internal control over financial reporting. 30 Table of Contents PART II. OTHER INFORMATION Item 1. Legal Proceedings None. Item 1A. Risk Factors An investment in our securities involves a high degree of risk. We operate in a dynamic and rapidly changing industry that involves numerous risks and uncertainties. The risks and uncertainties described below are not the only ones we face. Other risks and uncertainties, including those that we do not currently consider material, may impair our business. If any of the risks discussed below actually occur, our business, financial condition, operating results or cash flows could be materially adversely affected. This could cause the value of our securities to decline, and you may lose all or part of your investment. We have marked with an asterisk (*) those risk factors below that include a substantive change from or update to the risk factors included in our Annual Report on Form 10-K for the year ended December 31, 2025, which was filed with the SEC on February 26, 2026. Business and Operational Risks *Our success depends on our ability to manage our growth and execute our corporate strategy. We continue to pursue the corporate strategy we announced in 2024, which is focused on innovation, growth, and value commitment, which includes, among other things, the acceleration or discontinuation of certain programs, the continued expansion of our pipeline, updates to our commercial organizational model, and cost transformation. If we are unable to successfully execute our strategy, our business, financial condition and results of operations may be materially and adversely affected. As part of the strategy, we are continuing to advance VOXZOGO for the treatment of conditions beyond achondroplasia, including hypochondroplasia, idiopathic short stature, and Noonan syndrome. VOXZOGO addresses larger patient populations than most of our other products, and product candidates that we are currently developing or may license or acquire in the future may be intended for similarly larger patient populations than we have historically targeted. We are also expanding our pipeline through external innovation. In April 2026, we acquired Amicus Therapeutics, Inc. (Amicus) to expand and diversify our rare disease product portfolio (the Amicus Acquisition). In addition, in July 2025 we acquired Inozyme Pharma, Inc. (Inozyme) to strengthen our Enzyme Therapies portfolio, adding a late-stage enzyme replacement therapy, BMN 401 (formerly INZ-701). In order to continue the development of our product candidates and marketing of products with larger markets, we will need to continue expanding our operations. To manage expansion effectively, we need to continue to develop and improve our research and development capabilities, manufacturing and quality capacities, sales and marketing capabilities, financial and administrative systems and standard processes for global operations. Our staff, financial resources, systems, procedures or controls may be inadequate to support our operations and may increase our exposure to regulatory, competitive, and corruption risks and our management may be unable to manage successfully current or future market opportunities or our relationships with customers and other third parties. In addition, there is no guarantee that our corporate strategy will generate its expected benefits and the costs associated with implementing such strategy may be greater than anticipated. The execution of such strategy may also adversely affect our internal programs and initiatives as well as our ability to recruit and retain skilled and motivated personnel. If we are unable to execute on our corporate strategy or realize its expected benefits, then our business, operating results and financial condition may be materially and adversely affected. If we fail to develop new products and product candidates or compete successfully with respect to acquisitions, joint ventures, licenses or other collaboration opportunities, our ability to continue to expand our product pipeline and our growth and development would be impaired. Our future growth and development depend in part on our ability to successfully develop new products from our development activities. The development of biopharmaceutical products is very expensive and time intensive and involves a great degree of risk. The outcomes of research and development programs are inherently uncertain and may not result in the commercialization of any products. Our competitors compete with us to attract organizations for acquisitions, joint ventures, licensing arrangements or other collaborations. To date, several of our former and current product programs have been acquired through acquisitions and several of our former and current product programs have been developed through licensing or collaborative arrangements, such as ALDURAZYME, KUVAN and NAGLAZYME. These collaborations include licensing proprietary technology from, and other 31 Table of Contents relationships with, academic research institutions. Our future success will depend, in part, on our ability to identify additional opportunities and to successfully enter into partnering or acquisition agreements for those opportunities. If our competitors successfully enter into partnering arrangements or license agreements with academic research institutions, we will then be precluded from pursuing those specific opportunities. Because each of these opportunities is unique, we may not be able to find a substitute. Several pharmaceutical and biotechnology companies have already established themselves in the field of genetic diseases. These companies have already begun many drug development programs, some of which target diseases that we are also targeting or may target in the future, and have already entered into partnering and licensing arrangements with academic research institutions, reducing the pool of available opportunities. Universities and public and private research institutions also compete with us. While these organizations primarily have educational or basic research objectives, they may develop proprietary technology and acquire patents that we may need for the development of our product candidates. We have in the past attempted and may in the future attempt to license this proprietary technology, if available. These licenses may not be available to us on acceptable terms, if at all. If we are unable to compete successfully with respect to acquisitions, joint venture and other collaboration opportunities, we may be limited in our ability to develop new products and to continue to expand our product pipeline. *We have in the past and may in the future pursue acquisitions of other companies or businesses, which could divert our management’s attention, fail to achieve the anticipated benefits and/or expose us to other risks or difficulties. As part of our new corporate strategy, we have acquired, entered into agreements to acquire, and may continue to acquire, companies or businesses that we believe could complement, expand or enhance our product offerings. For example, in April 2026, we acquired Amicus to expand and diversify our rare disease product portfolio. In addition, in July 2025, we completed the acquisition of Inozyme to strengthen our Enzyme Therapies portfolio with BMN 401, which is currently being assessed for the treatment of ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) deficiency. Our acquisition strategy may divert the attention of management and cause us to incur various expenses in identifying, investigating and pursuing suitable acquisitions, whether or not such acquisitions are consummated. In addition, once an acquisition is closed, integrating our business practices and operations with the acquired business’ so that we can fully realize the anticipated benefits of the acquisition could require us to devote significant management attention and resources. The success of current and future acquisitions will depend, in part, on our ability to realize the anticipated benefits from successfully combining our and the acquired businesses’ operations. We may face risks or experience difficulties successfully integrating acquired businesses, such as Inozyme or Amicus, with our operations. Such difficulties could result in the failure to achieve revenue that we anticipate, the loss of key employees that may be difficult to replace in the very competitive pharmaceutical field, the failure to harmonize both companies’ corporate cultures, the disruption of each company’s ongoing businesses or inconsistencies in standards, controls, procedures and policies that adversely affect our ability to maintain relationships with suppliers, collaboration partners, clinical trial investigators or managers of our clinical trials. See “—Risks Related to the Amicus Acquisition” in this section for additional risks related to the Amicus Acquisition. Acquisitions could also result in dilutive issuances of equity securities, the incurrence of debt, contingent liabilities, amortization expenses, impairment of goodwill and/or purchased long-lived assets, and restructuring charges, any of which could adversely affect our operating results and financial condition. For example, we have incurred significant indebtedness in connection with the Amicus Acquisition. In addition, acquired product candidates, such as BMN 401, may not result in regulatory approval, may not perform as expected, may not be successful, may require significantly greater resources and investments than originally anticipated or may not produce the revenues, earnings or business synergies that we anticipated. As a result, the anticipated benefits of an acquisition may not be realized fully within the expected timeframe or at all or may take longer to realize or cost more than expected, which could materially and adversely affect our business, financial condition, results of operations and growth prospects. If we do not achieve our projected development goals in the timeframes we announce or fail to achieve such goals, the commercialization of our product candidates may be delayed or never occur and the credibility of our management may be adversely affected and, as a result, our stock price may decline. For planning purposes, we estimate the timing of the accomplishment of various scientific, clinical, regulatory and other product development goals, which we sometimes refer to as milestones. These milestones may include the commencement or completion of scientific studies and clinical trials and the submission of regulatory filings. From time to time, we publicly announce the expected timing of some of these milestones. All of these milestones are based on a variety of assumptions. The actual timing of these milestones can vary dramatically compared to our estimates or the milestones may never be achieved, in many cases for reasons beyond our control. If we do not meet development milestones as publicly announced, the commercialization of our products may be delayed or never occur and the credibility of our management may be adversely affected and, as a result, our stock price may decline. 32 Table of Contents *If we fail to compete successfully with respect to product sales, we may be unable to generate sufficient sales to recover our expenses related to the development of a product program or to justify continued marketing of a product and our revenues could be adversely affected. Our competitors may develop, manufacture and market products that are more effective or less expensive than ours. They may also obtain regulatory approvals for their products faster than we can obtain them (including those products with orphan drug designation, which may prevent us from marketing our product entirely for seven years, along with other regulatory exclusivities that could block approval) or commercialize their products before we do. With respect to VOXZOGO, we face competition from other companies that have developed, and may in the future develop, products for treatment for achondroplasia. As we commercialize our products, we have faced and may continue to face intense competition from other pharmaceutical companies, some of which may have more extensive resources and/or established relationships in the communities we seek to treat. If we do not compete successfully, our revenues would be adversely affected, and we may be unable to generate sufficient sales to recover our expenses related to the development of a product program or to justify continued marketing of a product. We also face competition from generic versions of our products. For example, generic versions of KUVAN are available in several countries around the world, including in the U.S. and the European Union (EU), which has adversely affected and will continue to adversely affect our revenues from KUVAN. Competitors launching generic versions of our products independently establish the price of such products and determine the types of discounts or rebates they will offer parties that purchase or pay for the product. Generic competition often results in decreases in the net prices at which branded products can be sold. After any introduction of a generic product, a significant percentage of the prescriptions written for our branded products will likely be filled with the generic product. Certain U.S. state laws allow for, and in some instances in the absence of specific instructions from the prescribing physician mandate, the dispensing of generic products rather than branded products when a generic version is available. We expect that the approval and launch of generic versions of our products and the approval and launch of branded products that compete with our products will continue to have a negative impact and could have a material adverse effect on our sales of our products and on our business, financial condition, results of operations and growth prospects. If we fail to obtain and maintain an adequate level of coverage and reimbursement for our products by third-party payers, the sales of our products would be adversely affected or there may be no commercially viable markets for our products. The course of treatment for patients using our products is expensive. We expect that most families of patients will not be capable of paying for our treatments themselves. For most of our products, we expect patients to need treatment for extended periods, and for some products throughout the lifetimes of the patients. There will be no commercially viable market for our products without coverage and reimbursement from third-party payers. Additionally, even if there is a commercially viable market, if the level of reimbursement is below our expectations, our revenues and gross margin will be adversely affected. Third-party payers, such as government or private healthcare insurers, carefully review and increasingly challenge the prices charged for drugs. Reimbursement rates from private companies vary depending on the third-party payer, the insurance plan and other factors. Obtaining coverage and adequate reimbursement for our products may be particularly difficult because of the higher prices often associated with drugs administered under the supervision of a physician. Reimbursement systems in international markets vary significantly by country and by region, and reimbursement approvals must be obtained on a country-by-country basis. Government authorities and other third-party payers are developing increasingly sophisticated methods of controlling healthcare costs, such as by limiting coverage and the amount of reimbursement for particular medications. Increasingly, third-party payers are requiring that drug companies provide them with predetermined discounts from list prices as a condition of coverage, are using restrictive formularies and preferred drug lists to leverage greater discounts in competitive classes, and are challenging the prices charged for medical products. Further, no uniform policy requirement for coverage and reimbursement for drug products exists among third-party payers in the U.S. Therefore, coverage and reimbursement for drug products can differ significantly from payer to payer. As a result, the coverage determination process is often a time-consuming and costly process that will require us to provide scientific and clinical support for the use of our products to each payer separately, with no assurance that coverage and adequate reimbursement will be applied consistently or obtained in the first instance. We cannot be sure that coverage and reimbursement will be available for any product that we commercialize or will continue to be available for any product that we have commercialized and, if reimbursement is available, what the level of reimbursement will be. Even if favorable coverage and reimbursement status is attained for one or more products for which we receive regulatory approval, less favorable coverage policies and reimbursement rates may be implemented in the future based on new legislation, the availability of alternative therapies and their pricing, coverage and reimbursement decisions by third-party payers, or other factors. Coverage and reimbursement may impact the demand for, or the price of, any product candidate for which we obtain marketing approval. If coverage and reimbursement are not available or reimbursement is available only to limited levels, we may not successfully commercialize any product candidate for which we obtain marketing approval or continue to market any product that has already been commercialized. 33 Table of Contents Reimbursement in the EU and many other territories must be negotiated on a country-by-country basis and in many countries the product cannot be commercially launched until pricing and/or reimbursement is approved. The timing to complete the negotiation process in each country is highly uncertain, and in some countries, we expect that it will exceed 12 months. Even after a price is negotiated, countries frequently request or require reductions to the price and other concessions over time. For our future products, we will not know what the reimbursement rates will be until we are ready to market the product and we actually negotiate the rates. If we are unable to obtain sufficiently high reimbursement rates for our products, they may not be commercially viable or our future revenues and gross margin may be adversely affected. Because the target patient populations for our products are relatively small, we must achieve significant market share and maintain high per-patient prices for our products to achieve and maintain profitability. All of our products target diseases with relatively small patient populations. As a result, our per-patient prices must be relatively high in order to recover our development and manufacturing costs and achieve and maintain profitability. For BRINEURA, NAGLAZYME and VIMIZIM in particular, we must market worldwide to achieve significant market penetration of the product. In addition, because the number of potential patients in each disease population is small, it is not only important to find patients who begin therapy to achieve significant market penetration of the product, but we also need to be able to maintain these patients on therapy for an extended period of time. Due to the expected costs of treatment for our products, we may be unable to maintain or obtain sufficient market share at a price high enough to justify our product development efforts and manufacturing expenses. Changes in methods of treatment of disease or failure of our products to gain acceptance by patients or the medical community could negatively impact demand for our products and adversely affect revenues. Even if our product candidates are approved, if doctors were to elect a course of treatment which does not include our products, this decision would reduce demand for our products and adversely affect revenues. For example, if gene therapy becomes widely used as a treatment of genetic diseases, the use of enzyme replacement therapy, such as ALDURAZYME, NAGLAZYME, and VIMIZIM in MPS diseases, could be greatly reduced. Changes in treatment method can be caused by the introduction of other companies’ products or the development of new technologies or surgical procedures which may not directly compete with ours, but which have the effect of changing how doctors decide to treat a disease. For example, we faced significant uncertainty as to whether gene therapy would gain the acceptance of the public or the medical community. In October 2025, we announced our plan to pursue options to divest ROCTAVIAN, including exploring out-licensing opportunities. Subsequently in December 2025, we committed to a plan to voluntarily withdraw ROCTAVIAN from the market due to lower than previously anticipated commercial opportunities. In connection with this strategic decision, we recorded approximately $240.0 million of restructuring charges in 2025 comprised of an inventory write-off, impairment of long-lived assets, severance and other cost. In addition, if we do not accurately forecast demand or manufacture products at levels in alignment with actual demand due to the failure of our products to gain acceptance by the patients or the medical community or other factors, then we may experience product shortages, pay a fee to contract manufacturers with whom we have non-cancellable capacity reservation agreements, or build excess inventory that may need to be written off, all of which could adversely affect our operating results. We have in the past entered and may in the future enter into licensing arrangements, and we may not realize the benefits of such licensing arrangements. We have in the past entered and may in the future enter into licensing arrangements with third parties. It is possible that we may not achieve financial or strategic benefits that justify a specific license, or we may otherwise not realize the benefits of such licensing arrangement. Further, licensing arrangements impose various diligence, milestone and royalty payment and other obligations on us. If we fail to comply with our obligations under any current or future licenses, our licensors may have the right to terminate these license agreements, which could harm our business prospects, financial condition and results of operations. Additionally, counterparties to our license agreements have in the past alleged and may in the future allege that we have breached a license agreement, which can result in litigation or other disputes that can divert management’s attention away from our business and require us to expend resources, as well as potentially having to negotiate new or reinstated licenses with less favorable terms. Any such situation could adversely affect our business, financial condition, and results of operations. Risks Related to the Amicus Acquisition *We may not realize the anticipated benefits from the Amicus Acquisition or accurately forecast the future performance of the combined company. The success of the Amicus Acquisition will depend, in part, on our ability to realize the anticipated benefits from successfully combining our and Amicus’ businesses. We plan on devoting substantial management attention and resources to integrating our and Amicus’ businesses so that we can fully realize the anticipated benefits of the Amicus Acquisition. This 34 Table of Contents integration process may be disruptive to our and Amicus’ businesses, and, if implemented ineffectively, could restrict realization of the expected benefits of the Amicus Acquisition. In addition, the acquired Amicus business, including GALAFOLD and POMBILITI + OPFOLDA, may not be successful, may require greater resources and investments than originally anticipated or may result in the assumption of unknown or contingent liabilities, which could have an adverse effect on us or our results of operations and our financial guidance. Potential difficulties we may encounter include the following: • the inability to successfully combine our and Amicus’ businesses in a manner that permits us to realize the anticipated benefits of the Amicus Acquisition in the timeframe currently anticipated or at all; • the failure to integrate internal systems, programs and internal controls, or applying different accounting policies, assumptions or judgments to Amicus’ operational results than Amicus applied in the past; • the inability to successfully obtain regulatory approval in new markets for, and continue to commercialize, GALAFOLD or POMBILITI + OPFOLDA on the currently anticipated timeline or at all; • the inability to effectively and efficiently integrate information technology and other systems; • issues not discovered as part of the transactional due diligence process or unanticipated liabilities or contingencies of Amicus, including employment or severance-related obligations under applicable law or other benefits arrangements, claims by or amounts owed to vendors or other commercial disputes, cyber incidents and information technology failures or delays, matters related to data privacy, data localization and the handling of personally identifiable information, intellectual property-related claims, including Hatch-Waxman litigation, and other unknown or contingent liabilities; • preserving the important licensing, marketing, and other commercial relationships of Amicus; • the complexities associated with managing the combined company; • the failure to retain key employees of either of the two companies who may be difficult to replace; • the disruption of each company’s ongoing businesses or inconsistencies in services, standards, controls, procedures and policies; • potential unknown liabilities and unforeseen increased expenses, delays or regulatory conditions associated with the Amicus Acquisition; and • performance shortfalls at one or both of the two companies as a result of the diversion of management’s attention caused by completing the Amicus Acquisition and integrating our and Amicus’ operations. Any of these risks could adversely affect our ability to maintain relationships with collaboration partners, vendors, employees and other commercial relationships or adversely affect our future operational results. As a result, the anticipated benefits of the Amicus Acquisition may not be realized or at all or may take longer to realize or cost more than expected, which could adversely affect our business, financial condition, including our ability to generate sufficient cash to service our indebtedness, including our 1.25% senior subordinated convertible notes due in 2027 (the 2027 Notes), our 5.5% senior unsecured notes due in 2034 (the 2034 Notes and together with the 2027 Notes, the Notes) and any loans outstanding under the 2026 Credit Agreement (as defined below), results of operations, growth prospects and our financial guidance, and we may be forced to take other actions to satisfy our obligations under our indebtedness, which may not be successful. In addition, changes in laws and regulations could adversely impact our business, financial condition, results of operations and growth prospects. Furthermore, the risks discussed above could also adversely affect our ability to accurately forecast the operational and financial performance of the combined company, including key financial metrics and prospects. If we are unable to accurately forecast and meet our financial guidance, our stock price can be materially adversely affected. *We have incurred and expect to incur material expenses related to the Amicus Acquisition. We have incurred and expect to incur material expenses in connection with the Amicus Acquisition and the subsequent integration of the business, operations, practices, policies and procedures of Amicus. These additional expenses could have an adverse effect on us or our results of operations. While we have assumed that a certain level of transaction and integration expenses would be incurred, there are a number of factors beyond our control that could affect the total amount or the timing of integration expenses. Many of the expenses that will be incurred, by their nature, are difficult to estimate accurately at the present time. 35 Table of Contents We may not realize the anticipated cost savings from the Amicus Acquisition. The benefits that we expect to achieve as a result of the Amicus Acquisition will depend, in part, on our ability to realize anticipated cost savings, including resulting from expected operational synergies and global infrastructure efficiencies. After the Amicus Acquisition, we believe that we will be able to, among other matters, save on our costs by being able to streamline administrative processes and harmonize global distribution of the two companies. Our success in realizing these cost savings, and the timing of this realization, depends on many factors. Even if we are able to consummate the Amicus Acquisition successfully, this may not result in the full realization of the cost savings that we currently expect, either within the expected timeframe, or at all. In addition, we cannot assure you that the costs to achieve these cost savings will not be higher than we anticipated. Therefore, we cannot assure you that any anticipated cost savings will be achieved or that our estimates and assumptions will prove to be accurate. If our cost savings are less than our estimates or our costs savings initiatives adversely affect our business or cost more or take longer to implement than we project, or if our assumptions prove to be inaccurate, our results could be lower than we anticipate. Our actual financial positions and results of operations may differ materially from the publicly filed unaudited pro forma condensed combined financial information. The unaudited pro forma condensed combined financial information contained in our Current Report on Form 8-K filed on January 26, 2026 is presented for illustrative purposes only and may differ materially from what our actual financial position or results of operations would have been had the Amicus Acquisition been completed on the dates indicated. The unaudited pro forma condensed combined financial information was derived from our and Amicus’ audited and unaudited historical financial statements and certain adjustments and assumptions have been made regarding the combined company after giving effect to the Amicus Acquisition and related transactions. The assets and liabilities of Amicus were measured at fair value based on various preliminary estimates using assumptions that we believed were reasonable utilizing information currently available. The process for estimating the fair value of acquired assets and assumed liabilities requires the use of judgment in determining the appropriate assumptions and estimates. These estimates may be revised as additional information becomes available and as additional analyses are performed. Differences between preliminary estimates in the unaudited pro forma condensed combined financial information and the final acquisition accounting will occur and could have a material impact on the unaudited pro forma condensed combined financial information and the ultimate combined company’s financial position and future results of operations. In addition, the assumptions used in preparing the unaudited pro forma condensed combined financial information may not prove to be accurate, and other factors may affect our financial condition or results of operations following the completion of the Amicus Acquisition. Regulatory Risks If we fail to obtain regulatory approval to commercially market and sell our product candidates, or if approval of our product candidates is delayed, we will be unable to generate revenues from the sale of these product candidates, our potential for generating positive cash flow will be diminished, and the capital necessary to fund our operations will increase. We must obtain regulatory approval to market and sell our product candidates. For example, in the U.S., we must obtain approval from the U.S. Food and Drug Administration (FDA) for each product candidate that we intend to commercialize, and in the EU, we must obtain approval from the European Commission (EC), based on the opinion of the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). The FDA and EC approval processes are typically lengthy and expensive, and approval is never certain. To obtain regulatory approval, we must first show that our product candidates are safe and effective for target indications through preclinical studies and clinical trials. Preclinical studies and clinical development are long, expensive and uncertain processes. Completion of clinical trials may take several years, and failure may occur at any stage of development. The length of time required varies substantially according to the type, complexity, novelty and intended use of a product candidate. Interim results of a preclinical test or clinical trial do not necessarily predict final results, and acceptable results in early clinical trials may not be repeated in later clinical trials. Accordingly, there are no assurances that we will obtain regulatory approval for any of our product candidates. Furthermore, there can be no assurance that approval of one of our product candidates by one regulatory authority will mean that other authorities will also approve the same product candidate. Similarly, in the EU, a positive CHMP opinion for approval of a product candidate does not guarantee that the EC will approve the product candidate. Moreover, regulatory authorities may approve a product candidate for fewer or more limited indications than requested. In addition, regulatory authorities may not approve the labeling claims that are necessary or desirable for the successful commercialization of our product candidates. We have had fewer interactions with regulatory authorities outside the U.S. and the EU as compared to our interactions with the FDA, the EC and the EMA. The approval procedures vary among countries and can involve additional clinical testing, and the time required to obtain approval may differ from that required to obtain FDA or EC approval. Moreover, clinical trials conducted in one country may not be accepted by regulatory authorities in other countries. Approval by the FDA or EC does not ensure 36 Table of Contents approval by regulatory authorities in other countries, and approval by one or more non-U.S. regulatory authorities does not ensure approval by regulatory authorities in other non-U.S. countries or by the FDA or EC. However, a failure or delay in obtaining regulatory approval in one country may have a negative effect on the regulatory process in others. The non-U.S. regulatory approval process may include all of the risks associated with obtaining FDA or EC approval. We may not obtain non-U.S. regulatory approvals on a timely basis, if at all. We may not be able to file for regulatory approvals and even if we file, we may not receive necessary approvals to commercialize our product candidates in any market. We also rely on independent third-party Contract Research Organizations (CROs) to file some of our non-U.S. marketing applications, and while we keep a close oversight on the activities we delegate to CROs, important aspects of the services performed for us by the CROs are out of our direct control. If we fail to adequately manage our CROs, if the CRO elects to prioritize work on our projects below other projects or if there is any dispute or disruption in our relationship with our CROs, the filing of our applications may be delayed. Although the FDA, the EC and the EMA have programs to facilitate expedited development and accelerated approval processes, the timelines agreed under legislative goals or mandated by regulations are subject to the possibility of substantial delays. Accordingly, even if any of our applications receives a designation to facilitate expedited development and accelerated approval processes, these designations may not result in faster review or approval for our product candidates compared to product candidates considered for approval under conventional procedures and, in any event, do not assure ultimate approval of our product candidates by regulatory authorities. In addition, the FDA, the EC, the EMA and other comparable international regulatory authorities have substantial discretion over the approval process for pharmaceutical products. These regulatory authorities may not agree that we have demonstrated the requisite level of product safety and efficacy to warrant approval and may require, and in the past have required, additional data. If we fail to obtain regulatory approval for our product candidates, we will be unable to market and sell those product candidates, which would have a negative effect on our business and financial condition. Regulatory authorities and the new requirements and guidelines they promulgate may lengthen the regulatory review process, require us to perform additional or larger studies, increase our development costs, lead to changes in regulatory positions and interpretations, delay or prevent approval and commercialization of our product candidates or lead to significant post-approval studies, limitations or restrictions. For example, the EU pharmaceutical reform will generally result in a decrease in data and market exclusivity in the EU. In addition, some of our product candidates are intended to be used in combination with a medical device, such as an injector or other delivery system. Some of these products intended to be used with a medical device may be regulated as “combination products” in the U.S. and the EU, which are generally defined as products consisting of components from two or more regulatory categories (e.g., drug/device, device/biologic, drug/biologic). In the U.S., each component of a combination product is subject to the requirements established by the FDA for that type of component, whether a new drug, biologic or device. In order to facilitate pre-market review of combination products, the FDA designates one of its centers to have primary jurisdiction for the pre-market review and regulation of the overall product based upon a determination by the FDA of the primary mode of action of the combination product. The determination whether a product is a combination product or two separately regulated products is made by the FDA on a case-by-case basis. In the EU, medical devices and medicinal products are regulated separately, through different legislative instruments. The related applicable requirements will vary depending on the type of drug-device combination product. If, for example, a device intended to administer a medicinal product is sold together with such medicinal product in such a way that they form a single integral product which is intended exclusively for use in the given combination and which is not reusable, that single integral product is regulated as a medicinal product. In addition, the relevant general safety and performance requirements (GSPRs) established for medical devices by EU medical devices legislation apply to the device component of such combination products. In addition, some of our products require use with an in vitro companion diagnostic. Our product candidates may also require use with an in vitro companion diagnostic if the FDA determines that the companion diagnostic is essential for safe and effective use of the product candidate. The FDA generally will require approval or clearance of the diagnostic, known as a companion diagnostic, at the same time that the FDA approves the therapeutic product. Most companion diagnostics require approval of a premarket approval application. In the EU, companion diagnostics are deemed to be in vitro diagnostic medical devices and must conform with the applicable GSPRs. To demonstrate compliance with the GSPRs, companion diagnostics must undergo a conformity assessment by a Notified Body. If the related medicinal product has been, or is in the process of being, authorized through the centralized procedure for the authorization of medicinal products, the Notified Body will, before it can issue the relevant EU technical documentation assessment certificate, be required to seek a scientific opinion from the EMA on the suitability of the companion diagnostic for use in relation to the medicinal product concerned. For medicinal products that have been or are in the process of authorization through any other route provided in EU legislation, the Notified Body must seek the opinion of the national competent authority of an EU Member State. Our product candidates intended for use with separately regulated devices, such as companion diagnostics, or expanded indications that we may seek for our products used with such devices, may not be approved or may be substantially delayed in receiving approval if the devices do not gain and/or maintain their own regulatory approvals, clearances, or certifications. Where approval of the drug or biologic product and device is sought under a single application, such as a drug with an injector or delivery system, the increased complexity of the review process may delay approval. The FDA and EU review processes and related criteria are complex, which could also lead to delays in the approval process. In addition, because these devices are provided by unaffiliated third-party companies, we are dependent on the sustained cooperation and effort of those third-party companies both to obtain regulatory approval and to maintain their own regulatory 37 Table of Contents compliance. Failure of third-party companies to assist in the approval process or to maintain their own regulatory compliance could delay or prevent approval of our product candidates, or limit our ability to sell a product once it is approved. From time to time during the development and regulatory approval process for our products and product candidates, we engage in discussions with the FDA, the EC, the EMA and other comparable international regulatory authorities regarding our development programs, including discussions about the regulatory requirements for approval. As part of these discussions, we sometimes seek advice in the design of our clinical programs from various regulatory authorities globally, but we do not always follow such guidance. This increases the chance of adverse regulatory actions, but we try to always provide appropriate scientific evidence to support approval. Moreover, sometimes different regulatory authorities provide different or conflicting advice. While we attempt to harmonize the advice we receive from multiple regulatory authorities, it is not always practical to do so. Also, we may choose not to harmonize conflicting advice when harmonization would significantly delay clinical trial data or is otherwise inappropriate. If we are unable to effectively and efficiently resolve and comply with the inquiries and requests of the FDA, the EC, the EMA and other comparable international regulatory authorities, the approval of our product candidates may be delayed and their value may be reduced. Any product for which we have obtained regulatory approval, or for which we obtain approval in the future, is subject to, or will be subject to, extensive ongoing regulatory requirements by the FDA, the EC, the EMA and other comparable international regulatory authorities, and if we fail to comply with regulatory requirements or if we experience unanticipated problems with our products, we may be subject to penalties, we will be unable to generate revenues from the sale of such products, our potential for generating positive cash flow will be diminished, and the capital necessary to fund our operations will be increased. Our marketed products have received regulatory approval to be commercially marketed and sold in the U.S., the EU, and certain other countries. Any product for which we have obtained regulatory approval, or for which we obtain regulatory approval in the future, along with the manufacturing processes and practices, post-approval clinical research, product labeling, advertising and promotional activities for such product, are subject to continual requirements of, and review by, the FDA, the EC, the EMA and/or other comparable international and national regulatory authorities. These requirements include submissions of safety and other post-marketing information and reports, registration and listing requirements, current Good Manufacturing Practices (cGMP) requirements relating to manufacturing, quality control, quality assurance and corresponding maintenance of records and documents, import and export requirements and record keeping. An example of the ongoing regulatory requirements our products are subject to is the PALYNZIQ Risk Evaluation and Mitigation Strategy (REMS) program. In the U.S., PALYNZIQ is only available through the REMS program, which is required by the FDA to mitigate the risk of anaphylaxis while using the product. Notable requirements of our REMS program include the following: • prescribers must be certified by enrolling in the REMS program and completing training; • prescribers must prescribe auto-injectable epinephrine with PALYNZIQ; • pharmacies must be certified with the REMS program and must dispense PALYNZIQ only to patients who are authorized to receive it; • patients must enroll in the REMS program and be educated about the risk of anaphylaxis by a certified prescriber to ensure they understand the risks and benefits of treatment with PALYNZIQ; and • patients must have auto-injectable epinephrine available at all times while taking PALYNZIQ. Failure of prescribers, pharmacies or patients to enroll in our REMS program or to successfully complete and comply with its requirements may result in regulatory action from the FDA or decreased sales of PALYNZIQ. The restrictions and requirements under our REMS program, as well as potential changes to these restrictions and requirements in the future, subject us to increased risks and uncertainties, any of which could harm our business. The requirement for a REMS program can materially affect the potential market for and profitability of a drug. We cannot predict whether the FDA will request, seek to require or ultimately require modifications to, or impose additional requirements on, the PALYNZIQ REMS program, or whether the FDA will permit modifications to the PALYNZIQ REMS program that we consider warranted. Any modifications required or rejected by the FDA could make it more difficult or expensive for us to distribute PALYNZIQ in the U.S., impair the safety profile of PALYNZIQ, disrupt continuity of care for PALYNZIQ patients and/or negatively affect sales of PALYNZIQ. In addition, in the EU, the marketing authorization for BRINEURA was granted under “exceptional circumstances”. As a result, the risk-benefit balance of BRINEURA is reviewed annually and the marketing authorization may be withdrawn if the risk-benefit ratio is no longer favorable. Failure to continue to show favorable risk-benefit balance for BRINEURA could result in the withdrawal of the marketing approval. Moreover, promotional communications with respect to drugs, including biologics, are subject to a variety of legal and regulatory restrictions and must be consistent with the information in the product’s approved labeling and Summary of Product Characteristics. In particular, a product may not be promoted for uses that are not approved by the FDA or the EC as reflected in 38 Table of Contents the product’s approved labeling. Although the FDA and other comparable international and national regulatory authorities do not regulate a physician’s choice of drug treatment made in the physician’s independent medical judgment, they do restrict promotional communications from companies or their sales force with respect to off-label uses of products for which marketing clearance has not been issued. The FDA and other national competent authorities or international regulatory authorities actively enforce the laws and regulations prohibiting the promotion of off-label uses, and a company that is found to have improperly promoted off-label uses may be subject to significant civil, criminal and administrative penalties. Thus, we are not able to promote any products we develop for indications or uses for which they are not approved. Additionally, in the EU, it is prohibited to promote prescription drugs to the general public and we are therefore limited to promote our products exclusively to healthcare professionals, which is also subject to restrictions. Public prosecutors, industry associations, healthcare professionals and other authorities and members of the public, including competitors, closely scrutinize advertising and promotion of any product in the EU. Moreover, if original FDA approval for one of our product candidates is granted via the accelerated approval pathway, we will be required to conduct a post-marketing confirmatory trial to verify and describe the clinical benefit in support of full approval. An unsuccessful post-marketing study or failure to complete such a study with due diligence could result in the withdrawal of the FDA’s marketing approval for a product candidate. For example, VOXZOGO is approved in the U.S. under accelerated approval based on an improvement in annualized growth velocity. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory studies. To fulfill this post-marketing requirement, we intend to use our ongoing open-label extension studies compared to available natural history. In addition, the FDA and the EC often require post-marketing testing and surveillance to monitor the effects of products. The FDA, the EC and other comparable international regulatory authorities may condition approval of our product candidates on the completion of such post-marketing clinical studies. These post-marketing studies may suggest that a product causes undesirable side effects or may present a risk to the patient. Discovery after approval of previously unknown problems with any of our products, manufacturers or manufacturing processes, or failure to comply with regulatory requirements, may result in actions such as: • the issuance of safety alerts, press releases or other communications containing warnings about related products; • modifications to promotional materials or corrective information to healthcare professionals; • restrictions on our ability to conduct clinical trials, including full or partial clinical holds on ongoing or planned trials; • suspensions or restrictions on our operations, including product manufacturing processes; • restrictions on the marketing of a product; • restrictions on product distribution; • requirements to conduct post-marketing clinical trials; • untitled or warning letters or other adverse publicity; • withdrawal of the products from the market; • suspended or withdrawn regulatory approvals; • refusal or delays to approve pending applications or supplements to approved applications that we submit; • recall of products; • refusal to permit the import or export of our products; • product seizure; • fines, restitution or disgorgement of profits or revenue; • injunctions; or • imposition of civil or criminal penalties. If such regulatory actions are taken, our value and our operating results will be adversely affected. Additionally, if the FDA, the EC or any other comparable international regulatory authorities withdraws its approval of a product, we will be unable to generate revenues from the sale of that product in the relevant jurisdiction, our potential for generating positive cash flow will be diminished and the capital necessary to fund our operations will be increased. Accordingly, we continue to expend significant time, money and effort in all areas of regulatory compliance, including manufacturing, production, product surveillance, post-marketing studies and quality control. To obtain regulatory approval to market our products, preclinical studies and costly and lengthy clinical trials are required and the results of the studies and trials are highly uncertain. Likewise, preliminary, initial or interim data from clinical trials should be considered carefully and with caution because the final data may be materially different from the preliminary, initial or interim data, particularly as more patient data become available. 39 Table of Contents As part of the drug development process, we must conduct, at our own expense, preclinical studies in the laboratory, including studies in animals, and clinical trials on humans for each product candidate. The number of preclinical studies and clinical trials that regulatory authorities require varies depending on the product candidate, the disease or condition the drug is being developed to address and regulations applicable to the particular drug. Generally, new drugs for diseases or conditions that affect larger patient populations, are less severe, or are treatable by alternative strategies must be validated through additional preclinical and clinical trials and/or clinical trials with higher enrollments. With respect to our early-stage product candidates, we may need to perform multiple preclinical studies using various doses and formulations before we can begin clinical trials, which could result in delays to our development timeline. Furthermore, even if we obtain favorable results in preclinical studies, the results in humans may be significantly different. After we have conducted preclinical studies, we must demonstrate that our product candidates are safe and efficacious for the intended indication and for use in the targeted human patients in order to receive regulatory approval for commercial sale. Clinical testing is expensive and can take many years to complete, and its outcome is inherently uncertain. Failure can occur at any time during the clinical trial process. The results of preclinical studies and early clinical trials of our product candidates may not be predictive of the results of later-stage clinical trials, and favorable data from interim analyses do not ensure the final results of a trial will be favorable. From time to time, we have published and may in the future publish or report preliminary, initial or interim data from our clinical trials. Preliminary, initial or interim data from our clinical trials may not be indicative of the final results of the trial and are subject to the risk that one or more of the clinical outcomes may materially change as patient enrollment continues and/or more patient data become available. In this regard, such data may show initial evidence of clinical benefit, but as patients continue to be followed and more patient data become available, there is a risk that any therapeutic effects will not be durable in patients and/or will decrease over time or cease entirely. Preliminary, initial or interim data also remain subject to audit and verification procedures that may result in the final data being materially different from such preliminary, initial or interim data. As a result, preliminary, initial or interim data should be considered carefully and with caution until the final data are available. Product candidates may fail to show the desired safety and efficacy traits despite having progressed through preclinical studies and initial clinical trials, or despite having favorable data in connection with an interim analysis. A number of companies in the biopharmaceutical industry have suffered significant setbacks in advanced clinical trials due to lack of efficacy or adverse safety profiles, notwithstanding promising results in earlier trials. Also, as noted above, we do not always follow the advice of regulatory authorities or comply with all of their requests regarding the design of our clinical programs. In those cases, we may choose a development program that is inconsistent with the advice of regulatory authorities, which may limit the jurisdictions where we conduct clinical trials and/or adversely affect our ability to obtain approval in those jurisdictions where we do not follow the regulatory advice. Adverse or inconclusive clinical results could stop us from obtaining regulatory approval of our product candidates. Additional factors that can cause delay or termination of our clinical trials include: • slow or insufficient patient enrollment; • slow recruitment of, and completion of necessary institutional approvals at, clinical sites; • budgetary constraints or prohibitively high clinical trial costs; • longer treatment time required to demonstrate efficacy; • lack of sufficient supplies of the product candidate; • adverse medical events or side effects in treated patients, including immune reactions; • lack of effectiveness of the product candidate being tested; • availability of competitive therapies to treat the same indication as our product candidates; • regulatory requests for additional clinical trials or preclinical studies; • deviations in standards for Good Clinical Practice (GCP); and • disputes with or disruptions in our relationships with clinical trial partners, including CROs, clinical laboratories, clinical sites, and principal investigators. Government price controls or other changes in pricing regulation could restrict the amount that we are able to charge for our current and future products, which would adversely affect our revenues and results of operations. We expect that pricing, coverage and reimbursement may be increasingly restricted in all the markets in which we sell our products. The escalating cost of healthcare has led to increased pressure on the healthcare industry to reduce costs. In particular, drug pricing by pharmaceutical companies has been under scrutiny for many years and continues to be subject to intense political and public debate in the U.S. and abroad. Governmental and private third-party payers have proposed healthcare reforms and cost reductions. A number of federal and state proposals to control the cost of healthcare, including the cost of drug treatments, have been made in the U.S. Specifically, there have been several U.S. congressional inquiries and proposed bills and enacted legislation designed to, among other things, bring more transparency to drug pricing, review the relationship between pricing and manufacturer patient programs, and reform government program reimbursement methodologies for drugs. Further, Congress and 40 Table of Contents