BIOMARIN PHARMACEUTICAL INC

CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE INCOME (LOSS)

Three and Nine Months Ended September 30, 2026 and 2025

(In thousands, except per share amounts)

CONDENSED CONSOLIDATED BALANCE SHEETS

September 30, 2026 and December 31, 2025

(In thousands, except share amounts)

CONDENSED CONSOLIDATED STATEMENTS OF STOCKHOLDERS’ EQUITY

Three and Nine Months Ended September 30, 2026 and 2025

(In thousands)

(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, 2025 filed with the SEC on February 26, 2026.

CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS

Nine Months Ended September 30, 2026 and 2025

(In thousands)

(1)BUSINESS OVERVIEW AND SIGNIFICANT ACCOUNTING POLICIES

Nature of Operations

BioMarin Pharmaceutical Inc. (the Company or BioMarin) is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with nine 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 unaudited 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 SEC for Quarterly Reports on Form 10-Q and do not include all of the information and note disclosures required by U.S. GAAP for complete financial statements, although management believes that the disclosures herein are adequate to ensure that the information presented is not misleading. The Condensed Consolidated Financial Statements should therefore be read in conjunction with the Consolidated Financial Statements and Notes thereto for the fiscal year ended December 31, 2025 included in the Company’s Annual Report on Form 10-K. The Condensed Consolidated Financial Statements include the accounts of the Company and its wholly owned subsidiaries. All intercompany transactions have been eliminated. The results of operations for the three and nine months ended September 30, 2026 are not necessarily indicative of the results that may be expected for the fiscal year ending December 31, 2026 or any other period.

Acquired IPR&D was previously included in Research and development (R&D) and is now presented separately in the Condensed Consolidated Statements of Comprehensive Income (Loss). The Company believes this presentation provides users of the financial statements with a better understanding of upfront and development milestone payments incurred to acquire IPR&D projects. Prior-period amounts have been reclassified to conform to the current presentation, resulting in a reclassification of $221.0 million from R&D expense to Acquired IPR&D for the three and nine months ended September 30, 2025. This reclassification had no impact on total operating expenses, net income (loss) or earnings (loss) per share.

On April 27, 2026, the Company completed the acquisition of Amicus Therapeutics, Inc. (Amicus). The results of Amicus’ operations, along with the preliminary estimated fair values of assets acquired and liabilities assumed in the acquisition, have been included in the Condensed Consolidated Financial Statements since the closing of the acquisition on April 27, 2026. Refer to Note 2 –Acquisitions for additional details related to the Amicus acquisition.

Management performed an evaluation of the Company’s activities through the date of filing of this Quarterly Report on Form 10-Q and has concluded that there were no subsequent events or transactions that occurred subsequent to the balance sheet date and prior to filing this Quarterly Report on Form 10-Q that would require recognition or disclosure in the Condensed Consolidated Financial Statements, except for the transaction disclosed in Note 15 to these Condensed Consolidated Financial Statements.

Use of Estimates

U.S. GAAP requires management to make estimates and assumptions that affect amounts reported in the Condensed Consolidated Financial Statements and accompanying disclosures. Although these estimates are based on management’s best knowledge of current events and actions that the Company may undertake in the future, actual results may be different from those estimates. The Condensed Consolidated Financial Statements reflect all adjustments of a normal, recurring nature that are, in the opinion of management, necessary for a fair presentation of results for these interim periods.

Significant Accounting Policies

There were no changes to the Company’s significant accounting policies during the nine months ended September 30, 2026, other than the updates as described below. The Company’s significant accounting policies are disclosed in Note 1 –Business Overview and Significant Accounting Policies to the Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025.

Foreign Currency

While the functional currency of the Company and certain subsidiaries continues to be the U.S. Dollar (USD), the functional currency of certain acquired foreign subsidiaries is their respective local currency.

For subsidiaries whose functional currency is the USD, monetary assets and liabilities denominated in foreign currencies are remeasured using period-end exchange rates and non-monetary assets and liabilities are remeasured using historical exchange rates. Foreign currency transaction gains and losses resulting from the remeasurement are recognized in Other Income (Expense), Net in the Consolidated Statements of Income.

For subsidiaries whose functional currency is their local currency, assets and liabilities are translated into USD using exchange rates in effect at the balance sheet date and income and expense items are translated using average foreign exchange rates for the period. Adjustments resulting from the translation of the financial statements of these certain foreign subsidiaries into USD are recorded in Accumulated Other Comprehensive Income (Loss), a separate component of stockholders' equity. Foreign currency transaction gains and losses arising from transactions denominated in a currency other than the subsidiary’s functional currency are recognized in Other Income (Expense), Net in the Consolidated Statements of Income.

Acquired IPR&D

Acquired IPR&D expenses are recorded when incurred and reflect costs of externally-developed IPR&D projects acquired directly in a transaction other than a business combination that do not have an alternative future use. These expenses include upfront and pre-commercialization milestone payments and the costs of rights to IPR&D projects. Subsequent development costs related to these projects are recognized as Research and development expense in the Consolidated Statements of Income as incurred.

Recent Accounting Pronouncements

There have been no new accounting pronouncements adopted by the Company or new accounting pronouncements issued by the Financial Accounting Standards Board (FASB) during the nine months ended September 30, 2026, as compared to the recent accounting pronouncements described in Note 1 to the Company’s Consolidated Financial Statements of the Company’s Annual Report on Form 10-K for the year ended December 31, 2025, that the Company believes are of significance or potential significance to the Company.

(2)ACQUISITIONS

Amicus Acquisition

On April 27, 2026, the Company completed the acquisition of Amicus, a publicly traded, global, biotechnology company. Under the terms of the merger agreement, the Company acquired all outstanding shares of Amicus common stock, and upon closing, Amicus became a wholly-owned subsidiary of BioMarin. The acquisition is expected to strengthen the Company's commercial portfolio by addingtwo new treatments to its existing portfolio of medicines that target lysosomal storage diseases: GALAFOLD (migalastat), the first oral treatment for Fabry disease, and POMBILITI (cipaglucosidase alfa-atga) + OPFOLDA (miglustat), a two-component therapy for Pompe disease. In connection with the acquisition, the Company also received U.S. rights to BMN 820 (also known as DMX-200), a potential first-in-class oral CCR2 inhibitor for focal segmental glomerulosclerosis (FSGS), a rare fatal kidney disease in Phase 3 development.

The total purchase consideration for the acquisition of Amicus was $5.3 billion, which consisted of cash consideration paid to former Amicus shareholders, cash payments related to the settlement of equity awards and stock options, the settlement of Amicus’ outstanding debt, and certain seller transaction costs paid by the Company on behalf of Amicus.

The transaction was financed through a combination of cash on hand and non-convertible debt financing. In connection with the acquisition, the Company issued $850.0 million in aggregate principal amount of 5.5% senior unsecured notes in February 2026, and obtained senior secured term loan facilities for $2.8 billion in aggregate principal in April 2026. Refer to Note 7 –Debt for additional details.

The transaction has been accounted for as a business combination using the acquisition method of accounting, which requires, among other things, that the assets acquired and liabilities assumed be recognized at their fair value on the acquisition date. The Company recorded acquisition-related costs of $32.8 million in Selling, General and Administrative (SG&A) on the Company’s Condensed Consolidated Statements of Comprehensive Income (Loss) during the nine months ended September 30, 2026.

Total consideration transferred for the acquisition of Amicus is summarized as follows:

(1)Cash settlement towards the stock options consists of $51.9 million paid for vested stock options and $11.0 million paid for the pre-combination portion of unvested stock options that were accelerated as part of the acquisition. The fair value of the unvested stock options attributable to the post-combination period of $13.0 million was recorded within SG&A on the Company’s Condensed Consolidated Statements of Comprehensive Income (Loss) during the second quarter of 2026.

(2)Includes cash settlement related to repayment of outstanding debt, prepayment penalty and accrued interest.

The following table summarizes the preliminary purchase price allocation of the assets acquired and liabilities assumed as of April 27, 2026, adjusted as of September 30, 2026:

(1) Includes measurement period adjustments primarily related to income tax during the third quarter of 2026 that increased deferred tax liabilities and goodwill by approximately $10.6 million.

(2) The Company expects to collect all accounts receivable based on information available as of the acquisition date.

The purchase price allocation presented above is preliminary as of September 30, 2026 and remains subject to revision during the measurement period, which extends up to one year from the acquisition date. The preliminary estimates of the fair values assigned to assets acquired and liabilities assumed, including identifiable intangible assets, deferred income taxes, and other acquired assets and assumed liabilities, are based on information available as of the filing date. As the Company obtains additional information and completes its valuation analyses, it may record adjustments to these preliminary amounts, with a corresponding adjustment to goodwill.

Intangible Assets

The fair values of the commercialized pharmaceutical products and IPR&D were determined using the multi-period excess earnings method. This approach determines fair value based on cash flow projections which are discounted to present value using a risk-adjusted rate of return. The discount rate used to determine the fair values of commercialized pharmaceutical products and IPR&D were 12.0% and 12.5%, respectively. These fair value measurements were based on significant inputs not observable in the market and thus represent Level 3 fair value measurements.

The following table presents details of the purchased intangible assets acquired:

The amortization expense related to acquired intangible assets for the three and nine months ended September 30, 2026 was $95.0 million and $164.0 million, respectively.

Inventory

The fair value of inventory acquired includes approximately $228.0 million step-up for GALAFOLD and POMBILITI + OPFOLDA. The fair value was primarily determined based on the estimated selling price of finished goods less the cost to complete the manufacturing process and selling effort. The amortization of the inventory step-up is recognized as a component of Cost of Sales on the Company’s Consolidated Statements of Comprehensive Income (Loss) as the inventory is sold. The amortization expense related to inventory step-up for the three and nine months ended September 30, 2026 was $17.5 million and $29.2 million, respectively.

Leases

The Company recorded lease liabilities of approximately $53.8 million, which represent the present value of remaining lease payments over the respective lease terms, with a corresponding right-of-use assets of approximately $46.1 million, which represent the estimated fair value based on current market rental rates. As of September 30, 2026, the right-of-use assets are presented in Other assets on the Company’s Condensed Consolidated Balance Sheets. As of September 30, 2026, the current portion of the lease liabilities is presented in Accounts Payable and Accrued Liabilities whereas the non-current portion is presented in Other Long-term Liabilities on the Company’s Condensed Consolidated Balance Sheets.

Goodwill

The recognized goodwill is primarily attributed to a deferred tax liability associated with the acquired intangible assets and inventory step-up. Goodwill recognized for Amicus is not deductible for tax purposes.

Results of Operations and Pro forma Financial Information

The following table presents revenues and net earnings for Amicus since the acquisition date of April 27, 2026 through September 30, 2026:

The unaudited pro forma results of operations for BioMarin as if the acquisition had occurred on January 1, 2025 are presented in the table below:

These unaudited pro forma results were based on estimates and assumptions that the Company believes are reasonable and are presented for informational purposes only. The unaudited pro forma financial information is not indicative of the results of operations that would have been achieved had the acquisition and related financing occurred at the beginning of fiscal year 2025. The unaudited pro forma information includes adjustments such as inventory step-up, amortization for intangible assets acquired, stock-based compensation expense, interest expense for acquisition financing, transaction costs and related income tax effects.

The significant nonrecurring adjustments reflected in the unaudited pro forma information above include the impact of (i) transaction costs of $47.1 million, (ii) post-combination stock-based compensation expense of $13.0 million, and (iii) commitment fee of $22.8 million relating to bridge loan commitment, all of which have been recognized as if incurred on the assumed acquisition date of January 1, 2025.

Alesta Acquisition

On August 31, 2026, the Company completed its acquisition of Alesta Therapeutics, B.V. (Alesta), a Dutch private limited liability company, committed to developing novel oral small-molecule therapies for underserved diseases, for a total consideration of $303.4 million. The terms of the acquisition also provide for regulatory and sales milestone payments of up to $215 million. Upon closing, Alesta 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, BMN 440 (formerly ALE1). BMN 440 is an orally active, small molecule for the potential treatment of hypophosphatasia (HPP), a rare genetic bone disease caused by mutations in the ALPL gene. 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 $282.3 million in acquired IPR&D expense related to BMN 440 IPR&D asset during the third quarter of 2026. The IPR&D charge is included in Acquired In-process Research and Development on the Company’s Condensed Consolidated Statements of Income.

The following summarizes the total consideration transferred and allocation to the assets acquired, liabilities assumed and acquired IPR&D:

(3)FINANCIAL INSTRUMENTS

The following tables show the Company’s cash, cash equivalents and available-for-sale securities by significant investment category as of the dates presented. There wereno marketable securities as of September 30, 2026 and all marketable securities as of December 31, 2025 were classified as available-for-sale.

(1) The Company’s short-term marketable securities as of December 31, 2025 matured inone year or less.

(2) The Company’s long-term marketable securities as of December 31, 2025 matured betweenone andfive years.

(4)SUPPLEMENTAL FINANCIAL STATEMENTS INFORMATION

Inventory consisted of the following:

Property, Plant and Equipment, Net consisted of the following:

Depreciation expense, net of amounts capitalized into inventory, for the three and nine months ended September 30, 2026 was $10.2 million and $28.6 million, respectively. 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.

Intangible Assets, Net consisted of the following:

Accounts Payable and Accrued Liabilities consisted of the following:

(5)FAIR VALUE MEASUREMENTS

The Company measures certain financial assets and liabilities at fair value in accordance with the policy described in Note 1 – Business Overview and Significant Accounting Policies to the Company’s Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025.

Other than the Company’s debt obligations disclosed in Note 7 –Debt, there wereno financial assets or liabilities that were remeasured using quoted prices in active markets for identical assets (Level 1) as of September 30, 2026 or December 31, 2025. Other than assets acquired from the Amicus acquisition discussed in Note 2 -Acquisitions, the Company hadno financial assets or liabilities that are remeasured on a recurring or non-recurring basis using unobservable inputs that reflect estimates and assumptions (Level 3) as of September 30, 2026 or December 31, 2025.

Level 2 assets and liabilities that are remeasured using significant observable inputs consisted of the following, except for derivatives, which are discussed in Note 6 –Derivative Instruments and Hedging Strategiesand the Term Loan A Facility, which is discussed in Note 7 -Debt:

There were no transfers between levels during the three and nine months ended September 30, 2026.

(6)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 USD, primarily the Euro. Certain of these forward contracts are designated as cash flow hedges and typically have maturities of up totwo 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 typically have maturities of up tothree 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.

The fair value of the Company’s derivatives, which are classified as Level 2 within the fair value hierarchy, were as follows:

For additional discussion of fair value measurements, see Note 5 - Fair Value Measurements.

The following tables summarize the impact of gains and losses from the Company's derivatives on its Condensed Consolidated Statements of Comprehensive Income (Loss) for the periods presented.

As of September 30, 2026, amounts expected to be reclassified from Accumulated Other Comprehensive Income (AOCI) to earnings over the next twelve months as the forecasted revenues and operating expense transactions occur were not material. For additional discussion of balances in AOCI see Note 8 –Accumulated Other Comprehensive Income.

(7)DEBT

As of September 30, 2026, the Company had outstanding fixed-rate notes and variable-rate term loans for an undiscounted aggregate principal amount of $4.2 billion. The debt detailed below incurs interest to be paid periodically in arrears. The following table summarizes information regarding the Company’s debt:

(1) For additional information related to the Company’s 2027 Notes, see Note 10 -Debt to the Company’s Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025.

(2) The fair value of the Company’s debt instruments are based on open-market trades (Level 1 of the fair value hierarchy), except for the Term Loan A Facility, whose fair value was based on observable market inputs for comparable debt instruments (Level 2 of the fair value hierarchy). For additional discussion of fair value measurements, see Note 1 – Business Overview and Significant Accounting Policies to the Company’s Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025.

Interest expense related to the Company’s debt and bridge loan commitment consisted of the following:

Term Loans and Revolving Credit Facility

In connection with the acquisition of Amicus in April 2026, the Company entered into a senior secured credit agreement (the 2026 Credit Agreement) consisting of an $800.0 million senior secured term loan A facility (Term Loan A Facility), a $2.0 billion senior secured term loan B facility (Term Loan B Facility, and, together with the Term Loan A Facility, the Term Facilities), and a $600.0 million senior secured revolving credit facility (the 2026 Revolving Facility). The proceeds from the Term Facilities were used to finance a portion of the acquisition consideration and related transaction costs. The Company incurred $46.2 million of issuance costs, which were deferred and will be amortized over the life of the Term Facilities and recorded as interest expense.

Borrowings under the Term Facilities and the 2026 Revolving Facility bear interest at variable rates based on Secured Overnight Financing Rate (SOFR) or an alternate base rate, plus an applicable margin as specified in the 2026 Credit Agreement. Interest on SOFR borrowings is payable at the end of each applicable interest period and, for interest periods greater than three months, at least quarterly.

The Term Loan A Facility matures on April 27, 2031 and requires quarterly principal repayments equal to 1.25% of the original principal amount, with the remaining outstanding principal balance due at maturity. The Term Loan B Facility matures on April 27, 2033 and requires quarterly principal repayments equal to 0.25% of the original principal amount, with the remaining outstanding principal balance due at maturity.

The 2026 Revolving Facility matures on April 27, 2031 and there wereno borrowings outstanding under the 2026 Revolving Facility as of September 30, 2026. The Company had the full $600.0 million of borrowing capacity available under the facility, subject to compliance with the terms of the 2026 Credit Agreement. The 2026 Credit Agreement contains customary affirmative and negative covenants, including, among others, covenants that restrict the Company's ability to incur additional indebtedness, create liens, make investments, pay dividends or make other restricted payments, dispose of assets, and enter into transactions with affiliates, in each case subject to exceptions set forth therein.

2034 Notes

In February 2026, the Company issued $850.0 million in aggregate principal amount of 5.5% senior unsecured notes with a maturity date of February 15, 2034, and the proceeds from the issuance were deposited into an escrow account and subsequently used to fund the Amicus acquisition. The 2034 Notes bear interest at the rate of 5.5% per annum. Interest is payable semi-annually with cash in arrears on February 15 and August 15 of each year, beginning August 15, 2026. Net proceeds from the offering were $832.3 million. The Company incurred $17.7 million of issuance costs, which were deferred and will be amortized over the life of the 2034 Notes and recorded as interest expense.

The indenture governing the Notes contains customary covenants that, among other things, restrict, with certain exceptions, the ability of the Company and its subsidiaries to incur additional debt, pay dividends, make certain other restricted payments, incur debt secured by liens, dispose of assets, engage in consolidations and mergers or sell or transfer all or substantially all of its assets. The offer and sale of the 2034 Notes have not been registered under the Securities Act or any state securities laws.

Bridge Commitment

In December 2025, the Company entered into a debt financing commitment letter and related fee letter with certain lenders, pursuant to which the lenders committed to provide the Company with debt financing up to approximately $3.7 billion (the Bridge Commitment) in the form of a 364-day senior secured bridge loan facility (Bridge Facility), the proceeds of which would be available for the acquisition of Amicus. In connection with the issuance of the 2034 Notes, the Bridge Commitment was reduced to $2.8 billion. Upon entry into the senior secured term loan facility in April 2026, the remaining Bridge Commitment was reduced tozero. Refer to Note 2 –Acquisitionsfor additional details related to the Amicus acquisition.

(8)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.

For additional discussion of reclassifications from AOCI see Note 6 – Derivative Instruments and Hedging Strategies.

(9)SEGMENT INFORMATION

The Company operates and is managed asone 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 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 (Loss). The CODM's analysis includes a comparison to budgeted results. Segment assets provided to the CODM are consistent with those reported on the Condensed Consolidated Balance Sheets with particular emphasis on the Company's available liquidity including cash, cash equivalents, investments, accounts receivable and inventory.

The following table includes information about segment revenue, significant segment expenses, and segment measure of profitability:

(1)Other segment expense, net during the three and nine months ended September 30, 2026 and 2025 includes intangible asset amortization, interest income and expense, other income, net and income tax expense.

The following table presents Total Revenues and disaggregates Net Product Revenues by product.

(1) The Company commenced recognizing revenue related to GALAFOLD and POMBILITI + OPFOLDA on April 27, 2026 following its acquisition of Amicus. Refer to Note 2 -Acquisitions for additional information.

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.

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.

Concentration Information

On a consolidated basis, one customer accounted for 20% of the Company’s September 30, 2026 accounts receivable balance compared to two customers who accounted for 20% and 10%, respectively, of the accounts receivable balance as of December 31, 2025. As of September 30, 2026, and December 31, 2025, the accounts receivable balance for Sanofi included $170.9 million and $148.0 million, respectively, of unbilled accounts receivable, which becomes payable to the Company when the product is sold through by Sanofi. The Company does not require collateral from its customers, but does perform periodic credit evaluations of its customers’ financial condition and requires prepayments in certain circumstances.

The Company is mindful that conditions in the current macroeconomic environment, such as inflation, changes in interest and foreign currency exchange rates, natural disasters, geopolitical instability, wars and military conflicts, impact of new or increased tariffs and escalating trade tensions, regulatory uncertainty, and supply chain disruptions, could affect the Company’s ability to achieve its goals. In addition, the Company sells its products in countries that face economic volatility and weakness. Although the Company has historically collected receivables from customers in such countries, sustained weakness or further deterioration of the local economies and currencies may cause customers in those countries to delay payment or be unable to pay for the Company’s products. The Company believes that the allowances for doubtful accounts related to these countries, if any, are adequate as of September 30, 2026 based on its analysis of the specific business circumstances and expectations of collection for each of the underlying accounts in these countries. The Company will continue to monitor these conditions and will attempt to adjust its business processes, as appropriate, to mitigate macroeconomic risks to its business.

(10)STOCK-BASED COMPENSATION

The Company has stockholder-approved equity incentive plans that provide for the granting of RSUs and stock options as well as other forms of equity compensation to its employees, officers and non-employee directors. The Company also has an Employee Stock Purchase Plan (ESPP). Compensation expense included in the Company’s Condensed Consolidated Statements of Comprehensive Income (Loss) for all stock-based compensation arrangements was as follows:

(11)RESTRUCTURING

Corporate Initiatives

In connection with the Company’s plan to simplify its organizational design and strategic initiatives, the Company committed to plans in the first quarter of 2026 to reduce its global workforce. Restructuring charges associated with the reorganization and strategic initiatives comprised of cash expenditures for severance and other employee termination benefits. These reductions are expected to be substantially complete by the end of 2026.

In connection with the Company’s corporate initiatives, the Company committed to a plan in the third quarter of 2026 to convert an office space to a laboratory, resulting in an asset impairment charge relating to building improvements.

Amicus

In April 2026, the Company committed to a restructuring plan related to the integration of Amicus. This restructuring plan is intended to realize certain targeted synergies following the acquisition, primarily through reductions in overlapping personnel costs within the general and administrative functions. Restructuring charges associated with Amicus integration comprised of cash expenditures for severance and other employee termination benefits. These reductions are expected to be completed in phases through April 2027.

Restructuring charges and adjustments were included in SG&A in the Condensed Consolidated Statements of Comprehensive Income (Loss). Restructuring charges consisted of the following:

The following unpaid balance as of September 30, 2026 was recorded to Accounts Payable and Accrued Liabilities on the Condensed Consolidated Balance Sheets:

(12)INCOME TAXES

The effective tax rates were 6.8% and (16.8)% for the three months ended September 30, 2026 and 2025, respectively, and 61.2% and 22.4% for the nine months ended September 30, 2026 and 2025, respectively. The effective tax rates for all periods reflected pre-tax losses resulting from nondeductible acquired IPR&D charges. The change in the effective tax rate for the three and nine months ended September 30, 2026 was primarily driven by the nondeductible acquired IPR&D charge having a greater impact on the estimated annual effective tax rate due to lower pre-tax income, which primarily reflected interest expense and amortization of acquired intangible assets related to the Amicus acquisition.

(13)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):

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):

(14)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.

License Agreements

As part of the Amicus acquisition, the Company assumed the existing license agreement with Dimerix Limited (Dimerix) for the U.S. commercialization rights for BMN 820 (also known as DMX-200) for the treatment of FSGS and other indications. Pursuant to the agreement, if future development, regulatory or commercial milestones are met, the Company will be obligated to pay Dimerix up to a maximum aggregate amount of $510.0 million with respect to FSGS and tiered royalties of BMN 820 net sales in the U.S. ranging from the low-teens to low-twenties. Refer to Note 2 - Acquisitions for additional information on the acquisition.

As of September 30, 2026, the Company was also subject to contingent payments, primarily comprised of development, regulatory and commercial milestones relating to certain other license agreements. Those considered reasonably possible totaled $485.5 million and, of this amount, the Company may pay up to $7.5 million over the next twelve months if certain contingencies are met.

Other Commitments

The Company uses experts and laboratories at universities and other institutions to perform certain R&D activities. These amounts are recorded as R&D expense as services are provided. In the normal course of business, the Company enters into various firm purchase commitments primarily to procure active pharmaceutical ingredients, certain inventory-related items and certain third-party R&D services, production services and facility construction services. As of September 30, 2026, such commitments were estimated at $768.8 million, of which $291.1 million is expected to be paid in 2026, with the remainder to be paid in 2027, 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.

(15)SUBSEQUENT EVENTS

In October 2026, the Company entered into an exclusive worldwide license agreement with GC Biopharma Corp. (GC Biopharma) and Hanmi Pharm Co., Ltd. (Hanmi) to develop and commercialize BMN 450 (also known as HM15421/GC1134A), an investigational long-acting enzyme replacement therapy for Fabry disease. Under the terms of the agreement, the Company will make an upfront payment of $70.0 million, which it expects to record as acquired IPR&D expense in the fourth quarter of 2026. GC Biopharma and Hanmi are also jointly eligible to receive up to an additional $405.0 million upon the completion of certain development and commercial milestones, and tiered royalties.

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).

Overview

We are a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, our San Rafael, California-based company has a proven track record of innovation, with nine 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.

During 2026, we completed the acquisitions of Amicus Therapeutics, Inc. (Amicus) and Alesta Therapeutics B.V. (Alesta). The accounting impacts of these transactions have been reflected in the Condensed Consolidated Financial Statements. Refer to Note 2 –Acquisitions for additional details related to these acquisitions.

A summary of our commercial products, as of September 30, 2026, is provided below:

(1) In 2026, we announced that we will no longer market ROCTAVIAN. For additional information related to ROCTAVIAN, see Note 19 - Restructuring to the Consolidated Financial Statements accompanying our Annual Report on Form 10-K for the year ended December 31, 2025.

(2) We acquired two commercial products, GALAFOLD and POMBILITI + OPFOLDA, in connection with the acquisition of Amicus on April 27, 2026. Refer to Note 2 -Acquisitions to our accompanying Condensed Consolidated Financial Statements for additional information.

Financial Highlights

Key components of our results of operations include the following:

See “Results of Operations” below for discussion of our results for the periods presented.

Uncertainty Relating to Macroeconomic Environment

Conditions in the current macroeconomic environment, such as inflation, changes in interest and foreign currency exchange rates, natural disasters, geopolitical instability, wars and military conflicts, impact of new or increased tariffs and escalating trade tensions, regulatory uncertainty, and supply chain disruptions, could impact our global revenue sources and our overall business operations. The extent and duration of such effects remain uncertain and difficult to predict. We are actively monitoring and managing our response and assessing actual and potential impacts to our operating results and financial condition, as well as developments in our business, which could further impact the developments, trends and expectations described below. See the risk factor, “Our business is affected by macroeconomic conditions.” described in “Risk Factors” in Part II, Item 1A of this Quarterly Report on Form 10-Q.

Recent Developments

We continued to grow our commercial business and advance our product candidate pipeline during 2026. We believe that the combination of our internal research programs, partnerships and acquisitions of external assets will allow us to continue to develop and commercialize innovative therapies for patients with serious and life-threatening rare diseases and medical conditions. We periodically conduct strategic portfolio assessment of research and development programs to determine which we believe have the strongest combination of scientific merit, opportunity for commercial success and potential value creation for stockholders. Based on such strategic portfolio assessments, certain programs that do not meet its threshold for further development and commercialization could be discontinued.

•In November 2026, we announced that the U.S. Food and Drug Administration (FDA) set a Prescription Drug User Fee Act (PDUFA) target action date of May 30, 2027 for supplemental new drug application (sNDA) to expand the use of VOXZOGO for the treatment of hypochondroplasia (HCH). We also announced that the European Medicines Agency (EMA) validated our submission for the approval of VOXZOGO to treat HCH.

•In October 2026, we entered into an exclusive worldwide license agreement with GC Biopharma Corp. (GC Biopharma) and Hanmi Pharm Co., Ltd. (Hanmi) to develop and commercialize BMN 450 (also known as HM15421/GC1134A), an investigational long-acting enzyme replacement therapy for Fabry disease. Under the terms of the agreement, we will make an upfront payment of $70.0 million that will be recorded as acquired IPR&D expense in the fourth quarter of 2026. GC Biopharma and Hanmi are also jointly eligible to receive up to an additional $405.0 million upon the completion of certain development and commercial milestones, and tiered royalties.

•In August 2026, we completed the acquisition of Alesta, a Dutch private limited liability company. The acquisition is intended to strengthen our Skeletal Conditions pipeline by adding BMN 440 (formerly ALE1), an orally active, small molecule for the potential treatment of hypophosphatasia (HPP). We accounted for this transaction as an asset acquisition since the lead asset, BMN 440, represents substantially all of the fair value of the gross assets acquired. See Note 2 to our accompanying Condensed Consolidated Financial Statements for additional information related to Alesta acquisition.

•In August 2026, we made the decision to discontinue development of BMN 401 across all indications.

•In July 2026, we announced that the FDA accepted our sNDA for full approval of VOXZOGO in children with achondroplasia, with a PDUFA target action date of February 28, 2027.

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.