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10-Q – 2025-10-28 – bmrn-20250930.htm
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QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2025 Or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to . 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: 192,114,344 shares of common stock, par value $0.001, outstanding as of October 23, 2025. 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 ® , KUVAN ® , NAGLAZYME ® , PALYNZIQ ® , 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. 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, 2024, which was filed with the Securities and Exchange Commission (the SEC) on February 24, 2025. You should carefully consider that information before you make an investment decision. 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 the Company’s management based on information currently available to management and should be considered in connection with any written or oral forward-looking statements that the Company may issue in the future as well as other cautionary statements the Company has made and may make. Except as required by law, the Company does 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. 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. • 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. Table of Contents 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 • 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. 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. • Global trade issues and changes in and uncertainties with respect to trade policies and export regulations, including import and export license requirements, trade sanctions, tariffs and international trade disputes, could adversely impact our business and operations, and reduce the competitiveness of our products and services relative to local and global competitors. 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 (Loss) (Unaudited) for the three and nine months ended September 30, 2025 and 2024 3 Condensed Consolidated Balance Sheets as of September 30, 2025 (Unaudited) and December 31, 2024 4 Condensed Consolidated Statement of Stockholders’ Equity (Unaudited) for the three and nine months ended September 30, 2025 and 2024 5 Condensed Consolidated Statements of Cash Flows (Unaudited) for the nine months ended September 30, 2025 and 2024 6 Notes to Condensed Consolidated Financial Statements (Unaudited) 7 Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations 21 Item 3. Quantitative and Qualitative Disclosures about Market Risk 32 Item 4. Controls and Procedures 32 PART II. OTHER INFORMATION 33 Item 1. Legal Proceedings 33 Item 1A. Risk Factors 33 Item 2. Unregistered Sales of Equity Securities and Use of Proceeds 63 Item 3. Defaults Upon Senior Securities 63 Item 4. Mine Safety Disclosures 63 Item 5. Other Information 63 Item 6. Exhibits 65 SIGNATURES 66 2 PART I. FINANCIAL INFORMATION Item 1. Financial Statements BIOMARIN PHARMACEUTICAL INC. CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE INCOME (LOSS) Three and Nine Months Ended September 30, 2025 and 2024 (In thousands, except per share amounts) (unaudited) Three Months Ended September 30, Nine Months Ended September 30, 2025 2024 2025 2024 REVENUES: Net product revenues $ 760,812 $ 733,867 $ 2,308,438 $ 2,073,811 Royalty and other revenues 15,321 11,873 38,250 32,791 Total revenues 776,133 745,740 2,346,688 2,106,602 OPERATING EXPENSES: Cost of sales 140,085 188,457 441,733 444,096 Research and development 409,478 184,901 729,517 573,675 Selling, general and administrative 268,415 253,480 706,810 742,418 Intangible asset amortization 4,847 5,009 14,540 33,606 Gain on sale of nonfinancial assets — — — ( 10,000 ) Total operating expenses 822,825 631,847 1,892,600 1,783,795 INCOME (LOSS) FROM OPERATIONS ( 46,692 ) 113,893 454,088 322,807 Interest income 17,854 18,053 55,694 57,203 Interest expense ( 2,579 ) ( 2,968 ) ( 8,121 ) ( 10,089 ) Other income, net 5,093 5,463 7,972 2,203 INCOME (LOSS) BEFORE INCOME TAXES ( 26,324 ) 134,441 509,633 372,124 Provision for income taxes 4,420 28,361 114,159 70,208 NET INCOME (LOSS) $ ( 30,744 ) $ 106,080 $ 395,474 $ 301,916 EARNINGS (LOSS) PER SHARE, BASIC $ ( 0.16 ) $ 0.56 $ 2.06 $ 1.59 EARNINGS (LOSS) PER SHARE, DILUTED $ ( 0.16 ) $ 0.55 $ 2.04 $ 1.56 Weighted average common shares outstanding, basic 192,032 190,429 191,639 189,806 Weighted average common shares outstanding, diluted 192,032 197,147 196,893 196,683 COMPREHENSIVE INCOME (LOSS) $ ( 20,116 ) $ 83,931 $ 299,884 $ 335,354 The accompanying notes are an integral part of these Condensed Consolidated Financial Statements. 3 BIOMARIN PHARMACEUTICAL INC. CONDENSED CONSOLIDATED BALANCE SHEETS September 30, 2025 and December 31, 2024 (In thousands, except share amounts) September 30, 2025 December 31, 2024 ⁽¹⁾ ASSETS (unaudited) Current assets: Cash and cash equivalents $ 1,250,108 $ 942,842 Short-term investments 227,731 194,864 Accounts receivable, net 790,266 660,535 Inventory 1,382,173 1,232,653 Other current assets 204,265 201,533 Total current assets 3,854,543 3,232,427 Noncurrent assets: Long-term investments 512,937 521,238 Property, plant and equipment, net 1,038,187 1,043,041 Intangible assets, net 233,112 255,278 Goodwill 196,199 196,199 Deferred tax assets 1,509,109 1,489,366 Other assets 270,781 251,391 Total assets $ 7,614,868 $ 6,988,940 LIABILITIES AND STOCKHOLDERS’ EQUITY Current liabilities: Accounts payable and accrued liabilities $ 798,438 $ 606,988 Total current liabilities 798,438 606,988 Noncurrent liabilities: Long-term convertible debt, net 596,663 595,138 Other long-term liabilities 163,056 128,824 Total liabilities 1,558,157 1,330,950 Stockholders’ equity: Common stock, $ 0.001 par value: 500,000,000 shares authorized; 192,098,751 and 190,761,349 shares issued and outstanding, respectively 192 191 Additional paid-in capital 5,900,968 5,802,068 Company common stock held by the Nonqualified Deferred Compensation Plan ( 11,291 ) ( 11,227 ) Accumulated other comprehensive income (loss) ( 33,937 ) 61,653 Retained earnings (accumulated deficit) 200,779 ( 194,695 ) Total stockholders’ equity 6,056,711 5,657,990 Total liabilities and stockholders’ equity $ 7,614,868 $ 6,988,940 (1) December 31, 2024 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, 2024, filed with the SEC on February 24, 2025. The accompanying notes are an integral part of these Condensed Consolidated Financial Statements. 4 BIOMARIN PHARMACEUTICAL INC. CONDENSED CONSOLIDATED STATEMENTS OF STOCKHOLDERS’ EQUITY Three and Nine Months Ended September 30, 2025 and 2024 (In thousands) (unaudited) Three Months Ended September 30, Nine Months Ended September 30, 2025 2024 2025 2024 Shares of common stock, beginning balances (1) 192,002 190,356 190,761 188,598 Issuances under equity incentive plans 97 198 1,338 1,956 Shares of common stock, ending balances 192,099 190,554 192,099 190,554 Total stockholders' equity, beginning balances (1) $ 6,027,113 $ 5,286,299 $ 5,657,990 $ 4,951,549 Common stock: Beginning balances (1) 192 190 191 189 Issuances under equity incentive plans, net of tax — 1 1 2 Ending balances 192 191 192 191 Additional paid-in capital: Beginning balances (1) 5,851,637 5,696,701 5,802,068 5,611,562 Issuances under equity incentive plans, net of tax ( 2,779 ) ( 2,688 ) ( 46,218 ) ( 33,031 ) Stock-based compensation 52,493 45,853 145,054 159,522 Change in Common stock held by the Nonqualified Deferred Compensation plan (NQDC) ( 383 ) 44 64 1,857 Ending balances 5,900,968 5,739,910 5,900,968 5,739,910 Company common stock held by the NQDC: Beginning balances (1) ( 11,674 ) ( 11,673 ) ( 11,227 ) ( 9,860 ) Common stock held by the NQDC 383 ( 44 ) ( 64 ) ( 1,857 ) Ending balances ( 11,291 ) ( 11,717 ) ( 11,291 ) ( 11,717 ) Accumulated other comprehensive income (loss): Beginning balances (1) ( 44,565 ) 26,799 61,653 ( 28,788 ) Other comprehensive income (loss) 10,628 ( 22,149 ) ( 95,590 ) 33,438 Ending balances ( 33,937 ) 4,650 ( 33,937 ) 4,650 Retained earnings (accumulated deficit) Beginning balances (1) 231,523 ( 425,718 ) ( 194,695 ) ( 621,554 ) Net income (loss) ( 30,744 ) 106,080 395,474 301,916 Ending balances 200,779 ( 319,638 ) 200,779 ( 319,638 ) Total stockholders' equity, ending balances $ 6,056,711 $ 5,413,396 $ 6,056,711 $ 5,413,396 (1) The beginning balances for the nine-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, 2024 filed with the SEC on February 24, 2025. The accompanying notes are an integral part of these Condensed Consolidated Financial Statements. 5 BIOMARIN PHARMACEUTICAL INC. CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS Nine Months Ended September 30, 2025 and 2024 (In thousands) (unaudited) Nine Months Ended September 30, 2025 2024 CASH FLOWS FROM OPERATING ACTIVITIES: Net income $ 395,474 $ 301,916 Adjustments to reconcile net income to net cash provided by operating activities: Depreciation and amortization 59,119 72,819 Non-cash interest expense 1,969 2,699 Accretion of discount on investments ( 3,695 ) ( 6,619 ) Stock-based compensation 134,496 149,652 Gain on sale of nonfinancial assets — ( 10,000 ) Impairment of assets and other noncash adjustments 2,967 19,889 Acquired in-process research & development expense 220,963 — Deferred income taxes 48,125 13,709 Unrealized foreign exchange gain 244 ( 22,352 ) Other ( 1,704 ) ( 1,254 ) Changes in operating assets and liabilities: Accounts receivable, net ( 107,378 ) ( 130,456 ) Inventory ( 98,796 ) ( 29,259 ) Other current assets ( 32,125 ) ( 19,939 ) Other assets ( 32,679 ) ( 31,839 ) Accounts payable and accrued liabilities 119,478 68,019 Other long-term liabilities 21,892 10,229 Net cash provided by operating activities 728,350 387,214 CASH FLOWS FROM INVESTING ACTIVITIES: Purchases of property, plant and equipment ( 62,316 ) ( 65,894 ) Maturities and sales of investments 294,547 478,436 Purchases of investments ( 313,262 ) ( 352,371 ) Proceeds from sale of nonfinancial assets — 10,000 Purchase of intangible assets ( 5,569 ) ( 11,225 ) Acquisition, net of cash acquired ( 285,193 ) — Other — 1,141 Net cash provided by (used in) investing activities ( 371,793 ) 60,087 CASH FLOWS FROM FINANCING ACTIVITIES: Proceeds from exercises of awards under equity incentive plans 7,714 41,415 Taxes paid related to net share settlement of equity awards ( 53,265 ) ( 72,651 ) Repayments of convertible debt — ( 494,987 ) Other — ( 3,083 ) Net cash used in financing activities ( 45,551 ) ( 529,306 ) Effect of exchange rate changes on cash ( 3,740 ) 2,326 NET INCREASE (DECREASE) IN CASH AND CASH EQUIVALENTS 307,266 ( 79,679 ) Cash and cash equivalents: Beginning of period $ 942,842 $ 755,127 End of period $ 1,250,108 $ 675,448 SUPPLEMENTAL CASH FLOW DISCLOSURES: Cash paid for interest $ 3,572 $ 6,654 Cash paid for income taxes $ 70,208 $ 36,088 SUPPLEMENTAL CASH FLOW DISCLOSURES FOR NON-CASH INVESTING AND FINANCING ACTIVITIES: Increase (decrease) in accounts payable and accrued liabilities related to fixed assets $ 7,014 $ 8,457 Increase (decrease) in accounts payable and accrued liabilities related to intangible assets $ ( 3,937 ) $ ( 9,059 ) 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. The San Rafael, California-based company, founded in 1997, has a proven track record of innovation, with eight commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin 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, 2024 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 and nine months ended September 30, 2025 are not necessarily indicative of the results that may be expected for the fiscal year ending December 31, 2025 or any other period. 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. 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. Significant Accounting Policies Other than as discussed below, there have been no changes to the Company’s significant accounting policies during the nine months ended September 30, 2025, 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, 2024. Acquisitions Acquisitions of businesses are accounted for using the acquisition method of accounting. The Company allocates the purchase price of acquired businesses to the tangible and intangible assets acquired and liabilities assumed based on their estimated fair values on the acquisition date. The purchase price allocation process requires management to make significant estimates and assumptions, especially at the acquisition date with respect to intangible assets and acquired in-process research and development (IPR&D) assets. Any excess of the purchase price over the estimated fair values of the net assets acquired is recognized as goodwill. If it is determined that the net assets acquired do not meet the definition of a business combination under the acquisition method of accounting, the transaction is accounted for as an asset acquisition and no goodwill is recognized. Acquired IPR&D assets with no alternative future use under the acquisition method of accounting are charged to Research and Development in the Consolidated Statements of Income. 7 Table of Contents BIOMARIN PHARMACEUTICAL INC. NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS (In thousands of U.S. Dollars, except per share amounts or as otherwise disclosed) Recent Accounting Pronouncements Other than as discussed below, 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 nine months ended September 30, 2025, 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, 2024, that the Company believes are of significance or potential significance to the Company. The following paragraphs discuss new accounting pronouncements issued by the FASB, but not yet adopted by the Company. Income Taxes In December 2023, the FASB issued ASU 2023-09, Income Taxes Topic 740 , Improvements to Income Tax Disclosures . The guidance requires disclosure of disaggregated information about the Company’s effective tax rate reconciliation as well as information on income taxes paid. The disclosure requirements will be applied on a prospective basis, with the option to apply it retrospectively. The effective date for the update is for fiscal years beginning after December 15, 2024. The Company is currently evaluating the effect of the update on the Company's related disclosures. Income Statement Disaggregation In November 2024, the FASB issued ASU 2024-03, Income Statement - Reporting Comprehensive Income Topic 220, Expense Disaggregation Disclosures . The guidance requires disclosure of additional information about specific expense categories in the notes to financial statements at interim and annual reporting periods. The disclosure requirements will be applied on a prospective basis, with the option to apply it retrospectively. The effective date for the update is for fiscal years beginning after December 15, 2026 and interim periods within fiscal years beginning after December 15, 2027. The Company is currently evaluating the effect of the update on the Company's related disclosures. Internal-use Software In September 2025, the FASB issued ASU 2025-06, Intangibles - Goodwill and Other - Internal-Use Software Topic 350-40: Accounting for and Disclosure of Software Costs . The guidance clarifies the accounting for costs related to internal-use software by removing all references to project stages and clarifying the threshold to be applied to begin capitalizing. The guidance also specifies that disclosure under Accounting Standards Codification Topic 360-10, Property, Plant, and Equipment - Overall , apply to capitalized software costs. The Company may apply the guidance using a prospective, retrospective or modified transition approach. The effective date for the update is for fiscal years beginning after December 15, 2027 and interim periods within fiscal years beginning after December 15, 2027. The Company is currently evaluating the effect of the update on the Company's related disclosures. (2) FINANCIAL INSTRUMENTS All marketable securities were classified as available-for-sale as of September 30, 2025 and December 31, 2024. The following tables show the Company’s cash, cash equivalents and available-for-sale securities by significant investment category as of the dates presented: 8 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) September 30, 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 $ 400,492 $ — $ — $ 400,492 $ 400,492 $ — $ — Level 2: Money market instruments 844,616 — — 844,616 844,616 — — Corporate debt securities 474,892 3,520 ( 26 ) 478,386 — 181,605 296,781 U.S. government agency securities 202,584 991 ( 21 ) 203,554 5,000 45,912 152,642 Asset-backed securities 63,313 423 ( 8 ) 63,728 — 214 63,514 Subtotal 1,585,405 4,934 ( 55 ) 1,590,284 849,616 227,731 512,937 Total $ 1,985,897 $ 4,934 $ ( 55 ) $ 1,990,776 $ 1,250,108 $ 227,731 $ 512,937 December 31, 2024 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 $ 329,619 $ — $ — $ 329,619 $ 329,619 $ — $ — Level 2: Money market instruments 613,223 — — 613,223 613,223 — — Corporate debt securities 503,202 2,410 ( 390 ) 505,222 — 168,104 337,118 Asset-backed securities 138,508 363 ( 344 ) 138,527 — 25,864 112,663 U.S. government agency securities 72,027 359 ( 33 ) 72,353 — 896 71,457 Subtotal 1,326,960 3,132 ( 767 ) 1,329,325 613,223 194,864 521,238 Total $ 1,656,579 $ 3,132 $ ( 767 ) $ 1,658,944 $ 942,842 $ 194,864 $ 521,238 (1) The Company’s short-term marketable securities mature in one year or less. (2) The Company’s long-term marketable securities mature between one and five years . As of September 30, 2025, the Company had the ability and intent to hold all investments that were in an unrealized loss position until maturity. The Company considered its intent and ability to hold the securities until recovery of amortized cost basis, the extent to which fair value is less than amortized cost basis, conditions specifically related to the security’s industry and geography, payment structure and history and changes to the ratings (if any) in determining that the decline in fair value compared to carrying value is not related to a credit loss. (3) SUPPLEMENTAL FINANCIAL STATEMENTS INFORMATION Inventory consisted of the following: September 30, 2025 December 31, 2024 Raw materials $ 115,302 $ 154,341 Work-in-process 781,170 550,678 Finished goods 485,701 527,634 Total inventory $ 1,382,173 $ 1,232,653 9 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) Property, Plant and Equipment, Net consisted of the following: September 30, 2025 December 31, 2024 Property, plant and equipment, gross $ 1,987,843 $ 1,934,832 Accumulated depreciation ( 949,656 ) ( 891,791 ) Total property, plant and equipment, net $ 1,038,187 $ 1,043,041 Depreciation expense, net of amounts capitalized into inventory, for the three and nine months ended September 30, 2025 was $ 10.6 million and $ 35.3 million, respectively. Depreciation expense, net of amounts capitalized into inventory, for the three and nine months ended September 30, 2024 was $ 10.9 million and $ 35.0 million, respectively. Intangible Assets, Net consisted of the following: September 30, 2025 December 31, 2024 Finite-lived intangible assets $ 722,742 $ 721,110 Accumulated amortization ( 489,630 ) ( 465,832 ) Net carrying value $ 233,112 $ 255,278 Accounts Payable and Accrued Liabilities consisted of the following: September 30, 2025 December 31, 2024 Accounts payable and accrued operating expenses $ 389,670 $ 235,403 Accrued compensation expense 177,755 202,513 Accrued rebates payable 140,049 120,835 Foreign currency exchange forward contracts 43,739 13,056 Accrued income taxes 22,606 12,567 Accrued royalties payable 6,945 7,923 Lease liability 7,578 7,574 Other 10,096 7,117 Total accounts payable and accrued liabilities $ 798,438 $ 606,988 (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, 2024. Other than the Company’s fixed-rate convertible 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 September 30, 2025 or 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) December 31, 2024. 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 September 30, 2025 or December 31, 2024. 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 : September 30, 2025 December 31, 2024 Assets: Other current assets: NQDC Plan assets $ 3,881 $ 2,928 Other assets: NQDC Plan assets 41,479 34,978 Restricted investments (1) 369 514 Total other assets 41,848 35,492 Total assets $ 45,729 $ 38,420 Liabilities: Accounts payable and accrued liabilities: NQDC Plan liability $ 3,881 $ 2,928 Other long-term liabilities: NQDC Plan liability 41,479 34,978 Total liabilities $ 45,360 $ 37,906 (1) The restricted investments as of September 30, 2025 and December 31, 2024 secure the Company’s irrevocable standby letters of credit obtained in connection with certain commercial agreements. There were no transfers between levels during the three and nine months ended September 30, 2025. (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 September 30, 2025 December 31, 2024 Derivatives designated as hedging instruments: Sell $ 1,098,405 $ 1,371,816 Purchase $ 258,318 $ 289,967 Derivatives not designated as hedging instruments: Sell $ 346,944 $ 344,101 Purchase $ 161,535 $ 63,617 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) 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 September 30, 2025 December 31, 2024 Derivatives designated as hedging instruments: Derivative Assets Other current assets $ 14,449 $ 60,192 Other assets 5,716 14,514 Subtotal $ 20,165 $ 74,706 Derivative Liabilities Accounts payable and accrued liabilities $ 42,918 $ 12,381 Other long-term liabilities 14,801 2,536 Subtotal $ 57,719 $ 14,917 Derivatives not designated as hedging instruments: Derivative Assets Other current assets $ 4,429 $ 4,934 Derivative Liabilities Accounts payable and accrued liabilities $ 821 $ 675 Total Derivative Assets $ 24,594 $ 79,640 Total Derivative Liabilities $ 58,540 $ 15,592 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, 2024. 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 September 30, Nine Months Ended September 30, 2025 2024 2025 2024 Derivatives Designated as Cash Flow Hedging Instruments Cash Flow Hedging Gains (Losses) Reclassified into Earnings Cash Flow Hedging Gains (Losses) Reclassified into Earnings Cash Flow Hedging Gains (Losses) Reclassified into Earnings Cash Flow Hedging Gains (Losses) Reclassified into Earnings Net product revenues $ ( 10,639 ) $ 3,365 $ ( 157 ) $ 5,617 Operating expenses $ 2,500 $ 172 $ 1,408 $ 486 Derivatives Not Designated as Hedging Instruments Gains (Losses) Recognized in Earnings Gains (Losses) Recognized in Earnings Gains (Losses) Recognized in Earnings Gains (Losses) Recognized in Earnings Operating expenses $ 945 $ ( 12,094 ) $ ( 35,234 ) $ 9,447 As of September 30, 2025, the Company expects to reclassify unrealized losses of $ 28.6 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 . 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) (6) DEBT Convertible Notes As of September 30, 2025, the Company had outstanding fixed-rate convertible notes for an undiscounted aggregate principal amount of $ 600.0 million. The convertible notes detailed below are senior subordinated convertible obligations, and interest is payable in arrears, semi-annually. The following table summarizes information regarding the Company’s convertible debt: September 30, 2025 December 31, 2024 1.25 % senior subordinated convertible notes due in May 2027 (the 2027 Notes) $ 600,000 $ 600,000 Unamortized discount net of deferred offering costs ( 3,337 ) ( 4,862 ) Total convertible debt, net $ 596,663 $ 595,138 Fair value of fixed-rate convertible debt (1) : 2027 Notes $ 569,841 $ 558,894 (1) The fair value of the Company’s fixed-rate convertible 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, 2024. Interest expense on the Company’s convertible debt consisted of the following: Three Months Ended September 30, Nine Months Ended September 30, 2025 2024 2025 2024 Coupon interest expense $ 1,875 $ 2,456 $ 5,625 $ 7,689 Accretion of discount on convertible notes 486 606 1,456 2,290 Amortization of debt issuance costs 27 67 80 364 Total interest expense on convertible debt $ 2,388 $ 3,129 $ 7,161 $ 10,343 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, 2024 for additional information related to the Company’s convertible debt. (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. 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) Three Months Ended September 30, 2025 Unrealized Gains (Losses) on Cash Flow Hedges Unrealized Gains (Losses) on Available-for-Sale Debt Securities Total AOCI balance as of June 30, 2025 $ ( 47,794 ) $ 3,229 $ ( 44,565 ) Other comprehensive income (loss) before reclassifications 1,972 674 2,646 Less: gain (loss) reclassified from AOCI ( 8,139 ) — ( 8,139 ) Tax effect — ( 157 ) ( 157 ) Net current-period other comprehensive income (loss) 10,111 517 10,628 AOCI balance as of September 30, 2025 $ ( 37,683 ) $ 3,746 $ ( 33,937 ) Three Months Ended September 30, 2024 Unrealized Gains (Losses) on Cash Flow Hedges Unrealized Gains (Losses) on Available-for-Sale Debt Securities Total AOCI balance as of June 30, 2024 $ 28,187 $ ( 1,388 ) $ 26,799 Other comprehensive income (loss) before reclassifications ( 26,099 ) 9,752 ( 16,347 ) Less: gain (loss) reclassified from AOCI 3,537 — 3,537 Tax effect — ( 2,265 ) ( 2,265 ) Net current-period other comprehensive income (loss) ( 29,636 ) 7,487 ( 22,149 ) AOCI balance as of September 30, 2024 $ ( 1,449 ) $ 6,099 $ 4,650 Nine Months Ended September 30, 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 ( 96,256 ) 2,513 ( 93,743 ) Less: gain (loss) reclassified from AOCI 1,251 — 1,251 Tax effect — ( 596 ) ( 596 ) Net current-period other comprehensive income (loss) ( 97,507 ) 1,917 ( 95,590 ) AOCI balance as of September 30, 2025 $ ( 37,683 ) $ 3,746 $ ( 33,937 ) Nine Months Ended September 30, 2024 Unrealized Gains (Losses) on Cash Flow Hedges Unrealized Gains (Losses) on Available-for-Sale Debt Securities Total AOCI balance as of December 31, 2023 $ ( 29,658 ) $ 870 $ ( 28,788 ) Other comprehensive income (loss) before reclassifications 34,312 6,826 41,138 Less: gain (loss) reclassified from AOCI 6,103 — 6,103 Tax effect — ( 1,597 ) ( 1,597 ) Net current-period other comprehensive income (loss) 28,209 5,229 33,438 AOCI balance as of September 30, 2024 $ ( 1,449 ) $ 6,099 $ 4,650 For additional discussion of reclassifications from AOCI see Note 5 – Derivative Instruments and Hedging Strategies . 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) (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. 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) The following table includes information about segment revenue, significant segment expenses, and segment measure of profitability: Three Months Ended September 30, Nine Months Ended September 30, 2025 2024 2025 2024 Total revenues $ 776,133 $ 745,740 $ 2,346,688 $ 2,106,602 Less: Cost of sales 140,085 188,457 441,733 444,096 R&D expenses Research and early pipeline 93,720 104,915 280,158 331,372 Later-stage clinical programs 251,650 10,078 279,074 17,792 Marketed products 64,108 69,908 170,285 224,511 SG&A expenses S&M expenses 131,327 108,945 355,958 351,103 G&A expenses 137,088 144,535 350,852 391,315 Other segment expense (income), net (1) ( 11,101 ) 12,822 73,154 44,497 Net income (loss) $ ( 30,744 ) $ 106,080 $ 395,474 $ 301,916 (1) Other segment expense, net during the three months ended September 30, 2025 and 2024 include intangible asset amortization, interest income and expense, other income (expense) and income tax expense. The following table presents Total Revenues and disaggregates Net Product Revenues by product. Three Months Ended September 30, Nine Months Ended September 30, 2025 2024 2025 2024 VOXZOGO $ 218,387 $ 189,785 $ 653,532 $ 526,597 VIMIZIM 182,755 178,158 586,525 548,694 NAGLAZYME 122,025 131,906 365,182 369,584 PALYNZIQ 108,778 90,634 307,985 254,634 ALDURAZYME 53,709 71,029 159,123 144,849 BRINEURA 47,953 37,005 137,007 121,361 KUVAN 24,025 28,115 76,195 92,576 ROCTAVIAN 3,180 7,235 22,889 15,516 Total net product revenues 760,812 733,867 2,308,438 2,073,811 Royalty and other revenues 15,321 11,873 38,250 32,791 Total revenues $ 776,133 $ 745,740 $ 2,346,688 $ 2,106,602 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 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 September 30, Nine Months Ended September 30, 2025 2024 2025 2024 United States $ 274,114 $ 235,515 $ 788,361 $ 655,963 Europe 195,184 191,017 667,900 607,755 Latin America 97,618 111,908 284,018 274,920 Rest of world 140,187 124,398 409,036 390,324 Total net product revenues marketed by the Company 707,103 662,838 2,149,315 1,928,962 ALDURAZYME net product revenues marketed by Sanofi 53,709 71,029 159,123 144,849 Total net product revenues $ 760,812 $ 733,867 $ 2,308,438 $ 2,073,811 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 September 30, Nine Months Ended September 30, 2025 2024 2025 2024 Customer A 14 % 11 % 15 % 13 % Customer B 12 10 11 10 Customer C 11 12 12 11 Total 37 % 33 % 38 % 34 % Concentration Information On a consolidated basis, one customer accounted for 23 % of the Company’s September 30, 2025 accounts receivable balance compared to December 31, 2024, when two customers accounted for 20 % and 11 % of the accounts receivable balance, respectively. As of September 30, 2025, and December 31, 2024, the accounts receivable balance for Sanofi included $ 135.7 million and $ 96.8 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, 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 September 30, 2025 based on its analysis of the specific business circumstances and expectations of collection for each of the underlying accounts in these countries. (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. 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) 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 September 30, Nine Months Ended September 30, 2025 2024 2025 2024 Cost of sales $ 4,281 $ 5,092 $ 10,628 $ 12,110 Research and development 14,676 11,496 40,648 45,108 Selling, general and administrative 30,308 26,901 83,220 92,434 Total stock-based compensation expense $ 49,265 $ 43,489 $ 134,496 $ 149,652 (10) EARNINGS (LOSS) 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. The following table sets forth the computation of basic and diluted earnings (loss) per common share (common shares in thousands): Three Months Ended September 30, Nine Months Ended September 30, 2025 2024 2025 2024 Numerator: Net income (loss), basic $ ( 30,744 ) $ 106,080 $ 395,474 $ 301,916 Add: Interest expense, net of tax, on the Company's convertible debt — 1,832 5,499 5,495 Net income (loss), diluted $ ( 30,744 ) $ 107,912 $ 400,973 $ 307,411 Denominator: Weighted-average common shares outstanding, basic 192,032 190,429 191,639 189,806 Effect of dilutive securities: Common stock issuable under the Company's equity incentive plans — 2,353 889 2,512 Common stock issuable under the Company’s convertible debt — 4,365 4,365 4,365 Weighted-average common shares outstanding, diluted 192,032 197,147 196,893 196,683 Earnings (loss) per common share, basic $ ( 0.16 ) $ 0.56 $ 2.06 $ 1.59 Earnings (loss) per common share, diluted $ ( 0.16 ) $ 0.55 $ 2.04 $ 1.56 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 September 30, Nine Months Ended September 30, 2025 2024 2025 2024 Common stock issuable under the Company's equity incentive plans 12,390 9,414 11,501 9,255 Common stock issuable under the Company’s convertible debt 4,365 — — — Total number of potentially issuable shares 16,755 9,414 11,501 9,255 18 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) (11) 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. Contingent Payments As of September 30, 2025, the Company was subject to contingent payments, primarily comprised of development, regulatory and commercial milestones. Those considered reasonably possible totaled $ 231.1 million, and the related contingencies are not expected to be met over the next 12 months. 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. The Company also has commitments related to enterprise resource planning (ERP) system implementation costs for which the Company is committed. As of September 30, 2025, such commitments were estimated at $ 549.0 million, of which $ 199.1 million is expected to be paid in 2025 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. (12) ASSET ACQUISITION On July 1, 2025, the Company completed its acquisition of Inozyme Pharma, Inc. (Inozyme), a publicly traded clinical-stage biopharmaceutical company dedicated to developing innovative therapeutics, for a total consideration of approximately $ 285 million, net of cash acquired. Upon closing, Inozyme became a wholly-owned subsidiary of BioMarin. The Company accounted for this transaction as an asset acquisition since substantially all of the fair value of the gross assets acquired was concentrated in a single identifiable IPR&D asset, INZ-701 (now known as BMN 401). The Company recognized the acquired assets and assumed liabilities based on the consideration paid, inclusive of transaction costs, on a relative fair value basis. As the acquired IPR&D does not have an alternative future use, the Company recorded approximately $ 221 million in acquired IPR&D expense related to the INZ-701 IPR&D asset during the third quarter of 2025. The IPR&D charge is included in Research and Development on the Company’s Condensed Consolidated Statements of Comprehensive Income (Loss). 19 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 summarizes the total consideration transferred and allocation to the assets acquired, liabilities assumed and acquired IPR&D: Cash consideration for outstanding shares $ 260,424 Cash consideration for equity awards 9,950 Consideration paid to Inozyme 270,374 Repayment of Inozyme debt 49,095 Employee-related incentive payments 2,714 Transaction costs 6,950 Total consideration $ 329,133 Cash and cash equivalents $ 43,939 Other assets 10,779 Deferred tax assets 68,697 Other liabilities ( 15,245 ) Total identifiable assets acquired, net 108,170 Acquired IPR&D 220,963 Total assets and liabilities $ 329,133 20 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). 21 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 eight 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 September 30, 2025, is provided below: Commercial Products Indication VOXZOGO (vosoritide) Achondroplasia VIMIZIM (elosulfase alpha) Mucopolysaccharidosis (MPS) IVA NAGLAZYME (galsulfase) MPS VI PALYNZIQ (pegvaliase-pqpz) Phenylketonuria (PKU) ALDURAZYME (laronidase) MPS I BRINEURA (cerliponase alfa) Neuronal ceroid lipofuscinosis type 2 (CLN2) KUVAN (sapropterin dihydrochloride) PKU ROCTAVIAN (valoctocogene roxaparvovec) Severe Hemophilia A Financial Highlights Key components of our results of operations include the following: Three Months Ended September 30, Nine Months Ended September 30, 2025 2024 2025 2024 Total revenues $ 776.1 $ 745.7 $ 2,346.7 $ 2,106.6 Cost of sales $ 140.1 $ 188.5 $ 441.7 $ 444.1 Research and development (R&D) $ 409.5 $ 184.9 $ 729.5 $ 573.7 Selling, general and administrative (SG&A) $ 268.4 $ 253.5 $ 706.8 $ 742.4 Provision for income taxes $ 4.4 $ 28.4 $ 114.2 $ 70.2 Net income (loss) $ (30.7) $ 106.1 $ 395.5 $ 301.9 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, 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 2025. 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. 22 Table of Contents Management’s Discussion and Analysis of Financial Condition and Results of Operations (continued) (In millions, except as otherwise disclosed) Based on such strategic portfolio assessments, certain programs that do not meet its threshold for further development and commercialization could be discontinued. In October 2025, we announced our plan to pursue options to divest ROCTAVIAN, including exploring out-licensing opportunities. We plan to continue to make ROCTAVIAN commercially available in the U.S., Italy and Germany until next steps are finalized. We will continue to provide support and monitoring for people treated with ROCTAVIAN. In September 2025, at the American Society for Bone and Mineral Research (ASBMR) Annual Meeting, we shared new data demonstrating improved spinal morphology – one of the factors that contributes to spinal stenosis, a leading cause of morbidity in achondroplasia – following treatment with VOXZOGO in children ages 5 and under. VOXZOGO is the only approved therapy with data showing a positive impact on spinal morphology, and these findings add to the extensive body of evidence supporting VOXZOGO’s health benefits beyond improving growth. In September 2025, we presented new data from the PALYNZIQ Phase 3 PEGASUS study in 12 to 17 year-olds demonstrating statistically significant blood phenylalanine (Phe) lowering compared to diet alone. In August 2025, we announced that Phase 1 data in its healthy volunteers study with BMN 333, BioMarin’s long-acting C-type natriuretic peptide (CNP), demonstrated area-under-the-curve (AUC) pharmacokinetic (PK) levels greater than three times the levels observed in other long-acting CNP studies, and no safety signals were noted. In July 2025, we completed the acquisition of Inozyme Pharma, Inc. (Inozyme), a publicly traded clinical-stage biopharmaceutical company dedicated to developing innovative therapeutics. The acquisition is intended to strengthen our enzyme therapies portfolio by adding a late-stage enzyme replacement therapy, BMN 401 (formerly INZ-701), for the treatment of ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) deficiency. We accounted for this transaction as an asset acquisition since the lead asset, BMN 401, represents substantially all of the fair value of the gross assets acquired. See Note 1 2 to our accompanying Condensed Consolidated Financial Statements for additional information related to Inozyme acquisition. 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 September 30, Nine Months Ended September 30, 2025 2024 Change 2025 2024 Change VOXZOGO $ 218.4 $ 189.9 $ 28.5 $ 653.5 $ 526.6 $ 126.9 Enzyme Therapies: VIMIZIM 182.8 178.2 4.6 586.5 548.7 37.8 NAGLAZYME 122.0 131.9 (9.9) 365.2 369.6 (4.4) PALYNZIQ 108.8 90.6 18.2 308.0 254.6 53.4 ALDURAZYME 53.7 71.0 (17.3) 159.1 144.8 14.3 BRINEURA 48.0 37.0 11.0 137.0 121.4 15.6 KUVAN 23.9 28.1 (4.2) 76.2 92.6 (16.4) ROCTAVIAN 3.2 7.2 (4.0) 22.9 15.5 7.4 Total net product revenues $ 760.8 $ 733.9 $ 26.9 $ 2,308.4 $ 2,073.8 $ 234.6 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 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) 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 September 30, 2025 as compared to the three months ended September 30, 2024 was primarily attributed to the following: • VOXZOGO and PALYNZIQ: 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 • NAGLAZYME: lower sales volume due to timing of orders in countries that place large government orders, primarily in Latin America. The increase in Net Product Revenues for the nine months ended September 30, 2025 as compared to the nine months ended September 30, 2024 was primarily attributed to the following: • VOXZOGO: higher sales volume from new patients initiating therapy across all regions; • PALYNZIQ: higher sales volume from new patients initiating therapy, primarily in the U.S.; and • VIMIZIM: higher sales volume from new patients initiating therapy across all regions and timing of orders in countries that place large government orders, primarily in Latin America. These increases were partially offset by the following: • KUVAN: lower product revenues attributed to increasing generic competition as a result of the loss of market exclusivity. 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. With respect to KUVAN, see also the risk factor “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” included in Part II, Item 1A of this Quarterly Report for additional information on risks we face. With respect to VOXZOGO, see also the risk factor “Our success depends on our ability to manage our growth and execute our corporate strategy.” 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, which we expect to continue in future periods. We 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, 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 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) Quarterly Report for additional information. The following table shows our Net Product Revenues denominated in USD and foreign currencies: Three Months Ended September 30, Nine Months Ended September 30, 2025 2024 Change 2025 2024 Change Sales denominated in USD $ 398.6 $ 378.9 $ 19.7 $ 1,160.6 $ 1,017.5 $ 143.1 Sales denominated in foreign currencies 362.2 355.0 7.2 1,147.8 1,056.3 91.5 Total net product revenues $ 760.8 $ 733.9 $ 26.9 $ 2,308.4 $ 2,073.8 $ 234.6 Three Months Ended September 30, Nine Months Ended September 30, 2025 2024 Change 2025 2024 Change Unfavorable impact of foreign currency exchange rates on product sales denominated in currencies other than USD $ (8.2) $ (22.6) $ 14.4 $ (28.9) $ (75.2) $ 46.3 The unfavorable impact for the three and nine months ended September 30, 2025 was primarily driven by weakening of the Argentine Peso, Brazilian Real and Mexican Peso, partially offset by strengthening of the Euro. The unfavorable impact for the three and nine months ended September 30, 2024 was primarily driven by weakening of Argentine Peso, Japanese Yen and Brazilian Real, partially offset by strengthening of the Euro. 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 valuation reserves. The following table summarizes our Cost of Sales and gross margin: Three Months Ended September 30, Nine Months Ended September 30, 2025 2024 Change 2025 2024 Change Total revenues $ 776.1 $ 745.7 $ 30.4 $ 2,346.7 $ 2,106.6 $ 240.1 Cost of sales $ 140.1 $ 188.5 $ (48.4) $ 441.7 $ 444.1 $ (2.4) Gross margin 81.9 % 74.7 % 7.2 % 81.2 % 78.9 % 2.3 % Cost of Sales decreased in the three months ended September 30, 2025 as compared to the three months ended September 30, 2024, primarily due to favorable product mix resulting from lower per-unit manufacturing costs and reduced ROCTAVIAN inventory reserves. Gross margin increased during the three and nine months ended September 30, 2025 as compared to the three and nine months ended September 30, 2024, primarily due to higher sales volume for products with higher margin in our Enzyme Therapies portfolio. 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. 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) 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. R&D expense consisted of the following: Three Months Ended September 30, Nine Months Ended September 30, 2025 2024 Change 2025 2024 Change Research and early pipeline $ 93.7 $ 104.9 $ (11.2) $ 280.1 $ 331.4 $ (51.3) Later-stage clinical programs 251.7 10.1 241.6 279.1 17.8 261.3 Marketed products 64.1 69.9 (5.8) 170.3 224.5 (54.2) Total R&D expense $ 409.5 $ 184.9 $ 224.6 $ 729.5 $ 573.7 $ 155.8 The increase in R&D expense for the three and nine months ended September 30, 2025 as compared to the three and nine months ended September 30, 2024 was primarily due to acquired In-Process Research and Development (IPR&D) related to a phase 3 clinical program from Inozyme acquisition and increased spend related to the continued progression of VOXZOGO indication for hypochondroplasia. These increases were partially offset by lower spend on Research and early pipeline due to discontinued programs and lower spend on ROCTAVIAN. 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. 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) SG&A expense consisted of the following: Three Months Ended September 30, Nine Months Ended September 30, 2025 2024 Change 2025 2024 Change S&M $ 131.3 $ 108.9 $ 22.4 $ 356.0 $ 351.1 $ 4.9 G&A 137.1 144.6 (7.5) 350.8 391.3 (40.5) Total SG&A expense $ 268.4 $ 253.5 $ 14.9 $ 706.8 $ 742.4 $ (35.6) S&M expense consisted of the following: Three Months Ended September 30, Nine Months Ended September 30, 2025 2024 Change 2025 2024 Change Enzyme Therapies $ 60.9 $ 52.2 $ 8.7 $ 165.5 $ 163.8 $ 1.7 VOXZOGO 46.1 28.0 18.1 123.5 90.4 33.1 ROCTAVIAN 8.3 17.0 (8.7) 22.1 65.0 (42.9) Other 16.0 11.7 4.3 44.9 31.9 13.0 Total S&M expense $ 131.3 $ 108.9 $ 22.4 $ 356.0 $ 351.1 $ 4.9 The increase in S&M expense for the three and nine months ended September 30, 2025 as compared to the three and nine months ended September 30, 2024 was primarily due to increased spending related to global expansion of VOXZOGO for achondroplasia and pre-launch activities of VOXZOGO for hypochondroplasia, partially offset by reduced activities related to ROCTAVIAN as we focused commercial efforts in the U.S., Germany and Italy to align with our updated ROCTAVIAN strategy announced during the second half of 2024. The decrease in G&A expense for the three and nine months ended September 30, 2025 as compared to the three and nine months ended September 30, 2024 was primarily due to severance and restructuring costs incurred in 2024 associated with our portfolio strategy review and the associated organizational redesign efforts that did not occur in 2025. The decrease in G&A expense was partially offset by post-acquisition severance charges related to Inozyme in 2025 and incremental administrative costs related to ongoing support of business initiatives during the year. Intangible Asset Amortization and Gain on Sale of Nonfinancial Assets Intangible Asset Amortization and Gain on Sale of Nonfinancial Assets were as follows: Three Months Ended September 30, Nine Months Ended September 30, 2025 2024 Change 2025 2024 Change Amortization of intangible assets $ 4.8 $ 5.0 $ (0.2) $ 14.5 $ 33.6 $ (19.1) Gain on sale of nonfinancial assets $ — $ — $ — $ — $ 10.0 $ (10.0) The decrease in Amortization of Intangible Assets for the nine months ended September 30, 2025 as compared to the nine months ended September 30, 2024 was due to the increase in the estimated useful life of an intangible asset as a result of the 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) extension of a patent during the second half of 2024 and an intangible asset becoming fully amortized during the fourth quarter of 2024. Gain on Sale of Nonfinancial Assets – in the first quarter of 2024, we recognized a gain of $10.0 million due to a third party’s achievement of a regulatory approval milestone related to previously sold intangible assets. 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 September 30, Nine Months Ended September 30, 2025 2024 Change 2025 2024 Change Interest income $ 17.9 $ 18.1 $ (0.2) $ 55.7 $ 57.2 $ (1.5) The change in Interest Income for the three and nine months ended September 30, 2025 as compared to the three and nine months ended September 30, 2024 was relatively flat. Interest Expense We incur interest expense primarily on our convertible debt. Interest Expense for the periods presented was as follows: Three Months Ended September 30, Nine Months Ended September 30, 2025 2024 Change 2025 2024 Change Interest expense $ 2.6 $ 3.0 $ (0.4) $ 8.1 $ 10.1 $ (2.0) The change in Interest Expense for the three and nine months ended September 30, 2025 as compared to the three and nine months ended September 30, 2024 was relatively flat. See Note 6 to our accompanying Condensed Consolidated Financial Statements for additional information regarding our debt. Other Income, Net Other Income, Net for the periods presented was as follows: Three Months Ended September 30, Nine Months Ended September 30, 2025 2024 Change 2025 2024 Change Other income, net $ 5.1 $ 5.5 $ (0.4) $ 8.0 $ 2.2 $ 5.8 The increase in Other Income, Net for the nine months ended September 30, 2025 as compared to the nine months ended September 30, 2024 was primarily due to proceeds from insurance related to damaged goods. The change in Other Income, Net for the three months ended September 30, 2025 as compared to the three months ended September 30, 2024 was relatively flat. 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) Provision for Income Taxes The Provision for Income Taxes for the periods presented was as follows: Three Months Ended September 30, Nine Months Ended September 30, 2025 2024 Change 2025 2024 Change Provision for income taxes $ 4.4 $ 28.4 $ (24.0) $ 114.2 $ 70.2 $ 44.0 The decrease in Provision for Income Taxes for the three months ended September 30, 2025 as compared to the three months ended September 30, 2024 was primarily due to lower pre-tax income, partially offset by unfavorable tax impact of a non-deductible acquired IPR&D charge in 2025. The increase in Provision for Income Taxes for the nine months ended September 30, 2025 as compared to the nine months ended September 30, 2024 was primarily due to higher pre-tax income and tax expense related to expiration of unexercised options in 2025. On July 4, 2025, the One Big Beautiful Bill (OBBB) Act, which includes a broad range of tax reform provisions, was signed into law in the United States. We have estimated the tax effects of the OBBB Act enacted during the quarter and included the related income tax benefit impact in our provision for income taxes. We will continue to evaluate the full impact of these legislative changes as additional guidance becomes available. Financial Condition, Liquidity and Capital Resources Our cash, cash equivalents, and investments as of September 30, 2025 and December 31, 2024 were as follows: September 30, 2025 December 31, 2024 Change Cash and cash equivalents $ 1,250.1 $ 942.8 $ 307.3 Short-term investments 227.7 194.9 32.8 Long-term investments 512.9 521.2 (8.3) Total cash, cash equivalents and investments $ 1,990.7 $ 1,658.9 $ 331.8 We believe cash generated from sales of our commercial products, in addition to our cash, cash equivalents and short-term investments will be sufficient to satisfy our liquidity requirements for at least the next 12 months. 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 and long-term investment balances. 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 convertible debt due in 2027, 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 convertible debt in cash. 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, 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. 29 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: Nine Months Ended September 30, 2025 2024 Change Net cash provided by operating activities $ 728.4 $ 387.2 $ 341.2 Net cash provided by (used in) investing activities $ (371.8) $ 60.1 $ (431.9) Net cash used in financing activities $ (45.6) $ (529.3) $ 483.7 The increase in net cash provided by operating activities in the nine months ended September 30, 2025 compared to September 30, 2024 was primarily attributed to an increase in net income adjusted for non-cash items, and timing of payments to vendors and cash receipts from our customers. The decrease in net cash provided by investing activities in the nine months ended September 30, 2025 compared to September 30, 2024 was primarily attributable to approximately $285 million net cash paid for acquisition of Inozyme and lower net maturities of available-for-sale securities. The decrease was partially offset by fewer purchases of fixed assets and intangible assets. The decrease in net cash used in financing activities in the nine months ended September 30, 2025 compared to September 30, 2024 was primarily due to $495 million settlement of the 2024 Notes that matured in August 2024. The decrease was also attributable to lower proceeds from exercises of equity awards in 2025. Financing Our $600.0 million (undiscounted) of convertible debt as of September 30, 2025 will impact our liquidity due to the semi-annual cash interest payments as well as the repayment of the principal amount, if not converted. As of September 30, 2025, our indebtedness consisted of our 1.25% senior subordinated convertible notes due in 2027, which, if not converted, will be required to be repaid in cash at maturity in May 2027. For additional information related to our convertible debt, see Note 6 to our accompanying Condensed Consolidated Financial Statements and Note 10 - Debt to the Consolidated Financial Statements accompanying our Annual Report on Form 10-K for the year ended December 31, 2024. In August 2024, we entered into an unsecured revolving credit facility providing for $600.0 million in revolving loan commitments. The credit facility is 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 credit facility matures in August 2029. As of September 30, 2025, there were no amounts outstanding under the credit facility and we were in compliance with all covenants. Material Cash Requirements Purchase Obligations As of September 30, 2025, we had obligations of approximately $549.0 million, of which $199.1 million is expected to be paid in 2025. 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. The amount also includes hosting fees and other enterprise resource planning (ERP) system implementation costs for which we are committed. Other Obligations As of September 30, 2025, we were subject to contingent payments considered reasonably possible of $231.1 million, and the related contingencies are not expected to be met over the next 12 months. See Note 11 to our accompanying Condensed Consolidated Financial Statements for additional discussion on our contingent obligations. Our lease commitments and unrecognized tax benefits as of September 30, 2025 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, 2024. See Note 11 to our accompanying Condensed Consolidated Financial Statements for additional information on our commitments. 30 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) 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 nine months ended September 30, 2025, 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, 2024, filed with the SEC on February 24, 2025. 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. 31 Table of Contents Item 3. Quantitative and Qualitative Disclosures About Market Risk Our market risks during the nine months ended September 30, 2025 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, 2024. 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 September 30, 2025. 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 ERP at certain subsidiaries, replacing existing operating and financial systems. The new ERP implementation is scheduled to occur in phases through 2026, with post-implementation activities following thereafter. 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 implementation and 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. 32 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, 2024, which was filed with the SEC on February 24, 2025. Business and Operational Risks *Our success depends on our ability to manage our growth and execute our corporate strategy. In 2024, we announced our new corporate strategy focused on innovation, growth, and value commitment, which includes, among other things, the acceleration or discontinuation of certain programs, the 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 have announced that we are advancing VOXZOGO for the treatment of conditions beyond achondroplasia, including hypochondroplasia, idiopathic short stature, Noonan syndrome, Turner syndrome, and SHOX deficiency. 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. 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 development of such 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 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 33 Table of Contents 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 will 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, and we may continue to acquire, companies or businesses that we believe could complement, expand or enhance our product offerings. For example, 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, 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. 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. 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. 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 34 Table of Contents that could block approval) or commercialize their products before we do. With respect to VOXZOGO, other companies are developing, and may in the future develop, products for treatment for achondroplasia that, if approved, could potentially compete with VOXZOGO even during the period of orphan drug exclusivity, for example by using an alternative formulation or a different delivery technology. 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. 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. 35 Table of Contents 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. We also face uncertainty as to whether gene therapy will gain the acceptance of the public or the medical community. The commercial success of ROCTAVIAN will depend, in part, on the acceptance of physicians, patients and third-party payers of gene therapy products in general, and our product in particular, as medically necessary, cost-effective and safe. In particular, our success will depend upon physicians prescribing our product in lieu of existing treatments they are already familiar with and for which greater clinical data may be available. Although administration of a gene therapy product like ROCTAVIAN is intended to correct an inborn genetic defect for at least several years, if the therapeutic effect of ROCTAVIAN decreases significantly or ceases entirely, it is uncertain whether redosing would be possible or effective. Adverse effects would not be able to be reversed or relieved by stopping dosing, and we may have to develop additional clinical safety procedures. Additionally, because the new gene copies are designed to reside permanently in a patient, there is a risk that they will disrupt other normal biological molecules and processes, including other healthy genes, and we may not learn the nature and magnitude of these side effects until long after clinical trials have been completed. Negative public opinion or more restrictive government regulations could have a negative effect on our business and financial condition and may delay or impair the successful commercialization of, and demand for, ROCTAVIAN. 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. For example, if we have ROCTAVIAN inventory in amounts that exceed actual demand, then such excess inventory may need to be written off, or we may incur an impairment charge with respect to the facility where ROCTAVIAN is manufactured, 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. Activist investor actions threatened or commenced against us have and could in the future cause us to incur substantial costs, divert management's attention and resources, cause uncertainty about the strategic direction of our business and adversely affect our business, financial position and results of operations. 36 Table of Contents We have been, and may in the future be, subject to activities initiated by activist investors. For example, in December 2023, we entered into a Cooperation Agreement with Elliott Investment Management L.P., Elliott Associates, L.P. and Elliott International, L.P., which expired in December 2024 pursuant to the terms of the agreement. We may not be successful in engaging constructively with one or more investors in the future despite our efforts to maintain constructive and ongoing communications with all investors. Resulting actions taken by activist investors from time to time have and could in the future conflict with our strategic direction, divert the attention of our Board of Directors, management, and employees, be costly and time-consuming, and disrupt the momentum in our business and operations, as well as our ability to execute our strategic plan. These types of actions may also create perceived uncertainties as to the future direction of our business or strategy, which may be exploited by our competitors and may make it more difficult to attract and retain qualified personnel, and may impact our relationships with investors, vendors, customers and other third parties. These types of actions could also impact the market price and the volatility of our common stock. In addition, we may choose to initiate, or may become subject to, litigation as a result of activist investor actions, which would serve as a further distraction to our Board of Directors, senior management and employees and could require us to incur significant additional costs. 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 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 37 Table of Contents 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, on April 26, 2023, the EC adopted a proposal for a new Directive and Regulation to revise the existing pharmaceutical legislation. While discussions are still ongoing as part of the legislative process, this proposal may result in a decrease in data and market exclusivity for our product candidates 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 a CE Certificate of Conformity, 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 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 38 Table of Contents 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 products have received regulatory approval to be commercially marketed and sold in the U.S., the EU, and certain other countries except ROCTAVIAN, which has received regulatory approval to be commercially marketed in the U.S. and conditional approval to be commercially marketed in the EU. 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. The conditional marketing authorization for ROCTAVIAN is, moreover, valid for one year and must be reviewed annually until all related conditions have been fulfilled to permit transfer to a full authorization. Failure to continue to show favorable risk-benefit balance for BRINEURA or satisfy the conditions related to ROCTAVIAN’s conditional marketing authorization could result in the withdrawal of the marketing approvals for these products. Moreover, promotional communications with respect to prescription 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 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. 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 39 Table of Contents 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. 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 40 Table of Contents 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 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 the executive branch have each indicated that they will continue to seek new legislative and/or administrative measures to control drug costs. In some international markets, the government controls the pricing, which can affect the profitability of drugs. Current government regulations and possible future legislation regarding healthcare may affect coverage and reimbursement for medical treatment by third-party payers, which may render our products not commercially viable or may adversely affect our future revenues and gross margins. International operations are also generally subject to extensive price and market regulations, and there are many proposals for additional cost-containment measures, including proposals that would directly or indirectly impose additional price controls or mandatory price cuts or reduce the value of our intellectual property portfolio. As part of these cost containment measures, some countries have imposed and continue to propose revenue caps limiting the annual volume of sales of our products. Some of these caps are significantly below the actual demand in certain countries, and if the trend regarding revenue caps continues, our future revenues and gross margins may be adversely affected. For example, in the EU, governments influence the price of medicinal products through their pricing and reimbursement rules and control of national healthcare systems that fund a large part of the cost of those products to consumers. EU Member States are free to restrict the range of medicinal products for which their national health insurance systems provide reimbursement and to control the prices of medicinal products for human use. Some jurisdictions operate positive and negative list systems under which products may only be marketed once a 41 Table of Contents