BIOMARIN PHARMACEUTICAL INC

Net Product Revenues

Net Product Revenues consisted of the following:

Net Product Revenues include revenues generated from our commercial products. In the U.S., our commercial products, except for PALYNZIQ and ALDURAZYME, are generally sold to specialty pharmacies or end users, such as hospitals, which act as retailers. PALYNZIQ is distributed in the U.S. through certain certified specialty pharmacies under the PALYNZIQ Risk Evaluation and Mitigation Strategy program, and ALDURAZYME is marketed worldwide by Sanofi. Outside the U.S., our commercial products are sold to authorized distributors or directly to government purchasers or hospitals, which act as the end users.

The increase in Net Product Revenues for the three months ended 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 1Aof 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 1Aof this

Quarterly Report for additional information. The following table shows our Net Product Revenues denominated in USD and foreign currencies:

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:

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.

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:

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:

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.

SG&A expense consisted of the following:

S&M expense consisted of the following:

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:

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 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.

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:

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:

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.

Provision for Income Taxes

The Provision for Income Taxes for the periods presented was as follows:

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:

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.

Our cash flows are summarized as follows:

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.

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.