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BIOMARIN PHARMACEUTICAL INC

For the period ended Mar 31, 2026. Filed May 5, 2026.

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A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.

Mar 31, 2026 · Filed May 5, 2026

BMRN$57.18+2.1%Stored close · as of Nov 9, 2026

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Item 1. Financial Statements

CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE INCOME

(In thousands, except per share amounts)

Three Months EndedMarch 31,
20262025
REVENUES:
Net product revenues$760,078$734,644
Royalty and other revenues6,13010,501
Total revenues766,208745,145
OPERATING EXPENSES:
Cost of sales194,999151,558
Research and development178,796158,731
Selling, general and administrative258,290206,116
Intangible asset amortization4,4834,847
Total operating expenses636,568521,252
INCOME FROM OPERATIONS129,640223,893
Interest income22,56019,013
Interest expense(14,958)(2,863)
Other income (expense), net3,961(1,954)
INCOME BEFORE INCOME TAXES141,203238,089
Provision for income taxes35,67652,403
NET INCOME$105,527$185,686
EARNINGS PER SHARE, BASIC$0.55$0.97
EARNINGS PER SHARE, DILUTED$0.54$0.95
Weighted average common shares outstanding, basic192,497190,967
Weighted average common shares outstanding, diluted197,671196,474
COMPREHENSIVE INCOME$114,763$143,184

CONDENSED CONSOLIDATED BALANCE SHEETS

March 31, 2026 and December 31, 2025

(In thousands, except share amounts)

March 31, 2026December 31, 2025 ⁽¹⁾
ASSETS · (unaudited)
Current assets:
Cash and cash equivalents$2,222,435$1,311,679
Short-term investments—248,930
Accounts receivable, net903,914908,214
Inventory1,273,2211,298,883
Other current assets205,500185,784
Total current assets4,605,0703,953,490
Noncurrent assets:
Long-term investments—492,242
Property, plant and equipment, net958,071952,508
Intangible assets, net204,662213,837
Goodwill196,199196,199
Deferred tax assets1,500,5981,508,697
Restricted cash equivalents850,000—
Other assets276,416277,049
Total assets$8,591,016$7,594,022
LIABILITIES AND STOCKHOLDERS’ EQUITY
Current liabilities:
Accounts payable and accrued liabilities$793,152$759,031
Total current liabilities793,152759,031
Noncurrent liabilities:
Long-term debt, net1,430,282597,176
Other long-term liabilities155,475150,816
Total liabilities2,378,9091,507,023
Stockholders’ equity:
Common stock, $0.001 par value: 500,000,000 shares authorized; 193,268,870 and 192,300,101 shares issued and outstanding, respectively193192
Additional paid-in capital5,966,8685,956,582
Company common stock held by the Nonqualified Deferred Compensation Plan(10,450)(10,508)
Accumulated other comprehensive loss(4,237)(13,473)
Retained earnings259,733154,206
Total stockholders’ equity6,212,1076,086,999
Total liabilities and stockholders’ equity$8,591,016$7,594,022

(1)December 31, 2025 balances were derived from the audited Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025, filed with the SEC on February 26, 2026.

CONDENSED CONSOLIDATED STATEMENTS OF STOCKHOLDERS’ EQUITY

(In thousands)

Three Months EndedMarch 31,
20262025
Shares of common stock, beginning balances (1)192,300190,761
Issuances under equity incentive plans969995
Shares of common stock, ending balances193,269191,756
Total stockholders' equity, beginning balances (1)$6,086,999$5,657,990
Common stock:
Beginning balances (1)192191
Issuances under equity incentive plans, net of tax11
Ending balances193192
Additional paid-in capital:
Beginning balances (1)5,956,5825,802,068
Issuances under equity incentive plans, net of tax(37,668)(49,333)
Stock-based compensation48,01241,617
Change in Common stock held by the Nonqualified Deferred Compensation plan (NQDC)(58)(50)
Ending balances5,966,8685,794,302
Company common stock held by the NQDC:
Beginning balances (1)(10,508)(11,227)
Common stock held by the NQDC5850
Ending balances(10,450)(11,177)
Accumulated other comprehensive income (loss):
Beginning balances (1)(13,473)61,653
Other comprehensive income (loss)9,236(42,502)
Ending balances(4,237)19,151
Retained earnings (accumulated deficit)
Beginning balances (1)154,206(194,695)
Net income105,527185,686
Ending balances259,733(9,009)
Total stockholders' equity, ending balances$6,212,107$5,793,459

(1)The beginning balances for the three-month periods were derived from the audited Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025 filed with the SEC on February 26, 2026.

CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS

(In thousands)

Three Months EndedMarch 31,
20262025
CASH FLOWS FROM OPERATING ACTIVITIES:
Net income$105,527$185,686
Adjustments to reconcile net income to net cash provided by operating activities:
Depreciation and amortization16,41122,069
Non-cash interest expense6,086660
Accretion of discount on investments(455)(1,362)
Stock-based compensation43,45837,700
Impairment of assets—2,967
Deferred income taxes9,22028,429
Unrealized foreign exchange losses (gains)6,710(10,026)
Other(5,374)(1,267)
Changes in operating assets and liabilities:
Accounts receivable, net(7,159)(57,590)
Inventory44,490(24,335)
Other current assets(11,551)(6,327)
Other assets1,484(1,624)
Accounts payable and accrued liabilities3,100(2,655)
Other long-term liabilities8,7042,069
Net cash provided by operating activities220,651174,394
CASH FLOWS FROM INVESTING ACTIVITIES:
Purchases of property, plant and equipment(20,923)(16,768)
Maturities and sales of investments767,27777,804
Purchases of investments(25,792)(89,274)
Other4,966—
Net cash provided by (used in) investing activities725,528(28,238)
CASH FLOWS FROM FINANCING ACTIVITIES:
Taxes paid related to net share settlement of equity awards(28,180)(38,779)
Proceeds from issuance of debt850,000—
Payments of debt issuance costs(8,653)—
Net cash provided by (used in) financing activities813,167(38,779)
Effect of exchange rate changes on cash1,410(1,416)
NET INCREASE IN CASH, CASH EQUIVALENTS AND RESTRICTED CASH EQUIVALENTS1,760,756105,961
Cash, cash equivalents and restricted cash equivalents:
Beginning of period$1,311,679$942,842
End of period$3,072,435$1,048,803
SUPPLEMENTAL CASH FLOW DISCLOSURES:
Cash paid for income taxes$14,644$10,388
SUPPLEMENTAL CASH FLOW DISCLOSURES FOR NON-CASH INVESTING AND FINANCING ACTIVITIES:
Accounts payable and accrued liabilities related to fixed assets$11,200$4,470
Accounts payable and accrued liabilities related to intangible assets$4,481$—

NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS (UNAUDITED)

(1)BUSINESS OVERVIEW AND SIGNIFICANT ACCOUNTING POLICIES

Nature of Operations

BioMarin Pharmaceutical Inc. (the Company or BioMarin) is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with a portfolio of commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, the Company seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients.

Basis of Presentation

These Condensed Consolidated Financial Statements have been prepared pursuant to U.S. generally accepted accounting principles (U.S. GAAP) and the rules and regulations of the Securities and Exchange Commission for Quarterly Reports on Form 10-Q and do not include all of the information and note disclosures required by U.S. GAAP for complete financial statements, although management believes that the disclosures herein are adequate to ensure that the information presented is not misleading. The Condensed Consolidated Financial Statements should therefore be read in conjunction with the Consolidated Financial Statements and Notes thereto for the fiscal year ended December 31, 2025 included in the Company’s Annual Report on Form 10-K. The Condensed Consolidated Financial Statements include the accounts of the Company and its wholly owned subsidiaries. All intercompany transactions have been eliminated. The results of operations for the three months ended March 31, 2026 are not necessarily indicative of the results that may be expected for the fiscal year ending December 31, 2026 or any other period.

Management performed an evaluation of the Company’s activities through the date of filing of this Quarterly Report on Form 10-Q and has concluded that there were no subsequent events or transactions that occurred subsequent to the balance sheet date and prior to filing this Quarterly Report on Form 10-Q that would require recognition or disclosure in the Condensed Consolidated Financial Statements, except for the transactions disclosed in Note 13 to these Condensed Consolidated Financial Statements.

Use of Estimates

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

Significant Accounting Policies

There have been no changes to the Company’s significant accounting policies during the three months ended March 31, 2026, as compared to the significant accounting policies disclosed in Note 1 –Business Overview and Significant Accounting Policies to the Company’s Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025.

Recent Accounting Pronouncements

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

(2)FINANCIAL INSTRUMENTS

All marketable securities were classified as available-for-sale as of March 31, 2026 and December 31, 2025.

The following tables show the Company’s cash, cash equivalents, restricted cash equivalents and available-for-sale securities by significant investment category as of the dates presented:

March 31, 2026
Amortized CostGrossUnrealizedGainsGrossUnrealizedLossesAggregate Fair ValueCash and Cash EquivalentsRestricted Cash Equivalents
Level 1:
Cash$352,248$—$—$352,248$352,248$—
Level 2:
Money market instruments2,720,187——2,720,1871,870,187850,000
Total$3,072,435$—$—$3,072,435$2,222,435$850,000
December 31, 2025
Amortized CostGrossUnrealizedGainsGrossUnrealizedLossesAggregate Fair ValueCash and Cash EquivalentsShort-termMarketableSecurities (1)Long-termMarketableSecurities (2)
Level 1:
Cash$415,760$—$—$415,760$415,760$—$—
Level 2:
Money market instruments895,919——895,919895,919——
Corporate debt securities472,5723,286(3)475,855—189,566286,289
U.S. government agency securities206,0181,197(1)207,214—59,115148,099
Asset-backed securities57,687420(4)58,103—24957,854
Subtotal1,632,1964,903(8)1,637,091895,919248,930492,242
Total$2,047,956$4,903$(8)$2,052,851$1,311,679$248,930$492,242

(1) The Company’s short-term marketable securities as of December 31, 2025 mature inone year or less, and were liquidated in the first quarter of 2026 to fund the Amicus Therapeutics, Inc. (Amicus) acquisition that closed in April 2026.

(2) The Company’s long-term marketable securities as of December 31, 2025 mature betweenone andfive years, and were liquidated in the first quarter of 2026 to fund the Amicus acquisition that closed in April 2026.

Restricted Cash Equivalents, Noncurrent

Restricted cash equivalents consists of funds held in escrow and restricted in their use to finance a portion of total consideration for the acquisition of Amicus. Refer to Note 13 –Subsequent Eventsfor additional details related to Amicus acquisition.

The following table provides a reconciliation of cash, cash equivalents, and restricted cash equivalents reported on the Company’s Condensed Consolidated Balance Sheets that sum to the total of the same such amounts shown in the Condensed Consolidated Statements of Cash Flows:

Cash and cash equivalents$2,222,435
Restricted cash equivalents850,000
Cash, cash equivalents and restricted cash equivalents$3,072,435

NOTES TO UNAUDITED CONDENSED CONSOLIDATED FINANCIAL STATEMENTS

(3)SUPPLEMENTAL FINANCIAL STATEMENTS INFORMATION

Inventory consisted of the following:

March 31,2026December 31,2025
Raw materials$97,850$106,510
Work-in-process1,054,613801,061
Finished goods120,758391,312
Total inventory$1,273,221$1,298,883

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

March 31,2026December 31,2025
Property, plant and equipment, gross$2,052,351$2,026,813
Accumulated depreciation(1,094,280)(1,074,305)
Total property, plant and equipment, net$958,071$952,508

Depreciation expense, net of amounts capitalized into inventory, for the three months ended March 31, 2026 and 2025 was $8.8 million and $14.1 million, respectively.

Intangible Assets, Net consisted of the following:

March 31,2026December 31,2025
Finite-lived intangible assets$722,360$723,966
Accumulated amortization(517,698)(510,129)
Net carrying value$204,662$213,837

Accounts Payable and Accrued Liabilities consisted of the following:

March 31,2026December 31,2025
Accounts payable and accrued operating expenses$395,469$312,768
Accrued compensation expense152,609219,422
Accrued rebates payable177,657166,925
Foreign currency exchange forward contracts31,67931,007
Accrued Interest9,460907
Lease liability9,3198,685
Accrued royalties payable6,6567,968
Accrued income taxes3,0103,667
Other7,2937,682
Total accounts payable and accrued liabilities$793,152$759,031

(4)FAIR VALUE MEASUREMENTS

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

Other than the Company’s fixed-rate debt disclosed in Note 6 –Debt, there wereno financial assets or liabilities that were remeasured using quoted prices in active markets for identical assets (Level 1) as of March 31, 2026 or December 31, 2025. The Company hadno financial assets or liabilities that are remeasured on a recurring basis using unobservable inputs that reflect estimates and assumptions (Level 3) as of March 31, 2026 or December 31, 2025.

Level 2 assets and liabilities that are remeasured using significant observable inputs consisted of the following, except for derivatives, which are discussed in Note 5 –Derivative Instruments and Hedging Strategies:

March 31,2026December 31,2025
Assets:
Other current assets:
NQDC Plan assets$4,055$3,765
Other assets:
NQDC Plan assets40,68741,689
Total assets$44,742$45,454
Liabilities:
Accounts payable and accrued liabilities:
NQDC Plan liability$4,055$3,765
Other long-term liabilities:
NQDC Plan liability40,68741,689
Total liabilities$44,742$45,454

There were no transfers between levels during the three months ended March 31, 2026.

(5)DERIVATIVE INSTRUMENTS AND HEDGING STRATEGIES

The Company uses foreign currency exchange forward contracts (forward contracts) to protect against the impact of changes in the value of forecasted foreign currency cash flows resulting from revenues and operating expenses denominated in currencies other than the U.S. Dollar (USD), primarily the Euro. Certain of these forward contracts are designated as cash flow hedges and have maturities of up 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 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.

Forward ContractsMarch 31,2026December 31,2025
Derivatives designated as hedging instruments:
Sell$1,488,793$1,573,184
Purchase$346,573$385,499
Derivatives not designated as hedging instruments:
Sell$424,423$356,285
Purchase$122,441$36,798

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

Balance Sheet LocationMarch 31,2026December 31,2025
Derivatives designated as hedging instruments:
Derivative Assets
Other current assets$25,563$17,585
Other assets7,6655,591
Subtotal$33,228$23,176
Derivative Liabilities
Accounts payable and accrued liabilities$30,538$30,134
Other long-term liabilities6,9029,905
Subtotal$37,440$40,039
Derivatives not designated as hedging instruments:
Derivative Assets
Other current assets$1,830$2,479
Derivative Liabilities
Accounts payable and accrued liabilities$1,141$873
Total Derivative Assets$35,058$25,655
Total Derivative Liabilities$38,581$40,912

For additional discussion of fair value measurements, see Note 1 –Business Overview and Significant Accounting Policies to the Company’s Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025.

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

Three Months Ended March 31,
20262025
Derivatives Designated as Cash Flow Hedging InstrumentsCash Flow Hedging Gains (Losses) Reclassified into Earnings
Net product revenues$(12,412)$12,545
Operating expenses$3,601$(2,606)
Derivatives Not Designated as Hedging InstrumentsGains (Losses) Recognized in Earnings
Operating expenses$187$(12,109)

As of March 31, 2026, the Company expects to reclassify unrealized losses of $5.0 million from Accumulated Other Comprehensive Income (AOCI) to earnings as the forecasted revenues and operating expense transactions occur over the next twelve months. For additional discussion of balances in AOCI see Note 7 –Accumulated Other Comprehensive Income.

(6)DEBT

As of March 31, 2026, the Company had outstanding fixed-rate notes for an undiscounted aggregate principal amount of $1.5 billion. The notes detailed below incur interest to be paid semi-annually in arrears. The following table summarizes information regarding the Company’s debt:

March 31,2026December 31,2025
5.5% senior unsecured notes due in February 2034 (the 2034 Notes)$850,000$—
Unamortized discount net of deferred offering costs(17,407)—
2034 Notes, net832,593—
1.25% senior subordinated convertible notes due in May 2027 (the 2027 Notes)$600,000$600,000
Unamortized discount net of deferred offering costs(2,311)(2,824)
2027 Notes, net597,689597,176
Long-term debt, net$1,430,282$597,176
Fair value of fixed-rate debt(1) :
2034 Notes$839,239$—
2027 Notes578,823576,267
Total fair value of fixed-rate debt$1,418,062$576,267

(1) The fair value of the Company’s fixed-rate long-term debt is based on open-market trades and classified as Level 1 in the fair value hierarchy. For additional discussion of fair value measurements, see Note 1 –Business Overview and Significant Accounting Policies to the Company’s Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025.

Interest expense on the Company’s long-term debt consisted of the following:

Three Months EndedMarch 31,
20262025
Coupon interest expense$8,805$1,875
Accretion of discount on convertible notes487485
Amortization of debt issuance costs27927
Total interest expense on debt$9,571$2,387

2034 Notes

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

The indenture governing the Notes contains customary covenants that, among other things, restrict, with certain exceptions, the ability of the Company and its subsidiaries to incur additional debt, pay dividends, make certain other restricted payments, incur debt secured by liens, dispose of assets, engage in consolidations and mergers or sell or transfer all or substantially all of its assets. The offer and sale of the 2034 Notes have not been registered under the Securities Act or any state securities laws and were offered only to qualified institutional buyers as defined in Rule 144A under the Securities Act.

See Note 10 - Debt to the Company’s Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025 for additional information related to the Company’s 2027 Notes and existing credit facility entered into in August 2024.

Bridge Commitment

In December 2025, the Company entered into a debt financing commitment letter and related fee letter with certain lenders, pursuant to which the lenders committed to provide the Company with debt financing up to approximately $3.7 billion (the Bridge Commitment) in the form of a 364-day senior secured bridge loan facility (Bridge Facility), the proceeds of which would be available for the acquisition of Amicus.

In connection with the issuance of the 2034 Notes, the Bridge Commitment was reduced to $2.8 billion. As a result, the Company recognized approximately $5.3 million of commitment fees during the three months ended March 31, 2026 and that is presented as Interest expense on the Condensed Consolidated Statements of Comprehensive Income. As of March 31, 2026, approximately $17.5 million in commitment fees were deferred and included in Other Current Assets on the Condensed Consolidated Balance Sheets. Refer to Note 13 –Subsequent Eventsfor additional details related to the Amicus acquisition.

(7)ACCUMULATED OTHER COMPREHENSIVE INCOME

The following tables summarize changes in the accumulated balances for each component of AOCI, including current-period other comprehensive income and reclassifications out of AOCI, for the periods presented.

Three Months Ended March 31, 2026
Unrealized Gains(Losses) on CashFlow HedgesUnrealized Gains(Losses) onAvailable-for-SaleDebt SecuritiesTotal
AOCI balance as of December 31, 2025$(17,244)$3,771$(13,473)
Other comprehensive income (loss) before reclassifications4,196(4,895)(699)
Less: gain (loss) reclassified from AOCI(8,811)—(8,811)
Tax effect—1,1241,124
Net current-period other comprehensive income (loss)13,007(3,771)9,236
AOCI balance as of March 31, 2026$(4,237)$—$(4,237)
Three Months Ended March 31, 2025
Unrealized Gains(Losses) on CashFlow HedgesUnrealized Gains(Losses) onAvailable-for-SaleDebt SecuritiesTotal
AOCI balance as of December 31, 2024$59,824$1,829$61,653
Other comprehensive income (loss) before reclassifications(33,652)1,434(32,218)
Less: gain (loss) reclassified from AOCI9,939—9,939
Tax effect—(345)(345)
Net current-period other comprehensive income (loss)(43,591)1,089(42,502)
AOCI balance as of March 31, 2025$16,233$2,918$19,151

For additional discussion of reclassifications from AOCI see Note 5 – Derivative Instruments and Hedging Strategies.

(8)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 research and development (R&D) organization is responsible for research and discovery of new product candidates and supporting the development and registration efforts for potential new products. The Company’s technical operations group is responsible for the development of manufacturing processes, supplying clinical drug product, and the manufacturing and distribution of the Company’s commercial products. The Company is also supported by corporate staff functions.

The Company’s Chief Executive Officer, as the Chief Operating Decision Maker (CODM), manages and allocates resources to the operations of the total company by assessing the overall level of resources available and how to best allocate them to support the Company’s long-term company-wide strategic goals. In making this decision, the CODM uses consolidated financial information for the purposes of evaluating performance, allocating resources, setting incentive compensation targets and planning and forecasting for future periods.

The key measure of segment profit or loss used by the CODM to allocate resources and assess the Company's performance is its Consolidated Net Income, as reported on the Condensed Consolidated Statements of Comprehensive Income. The CODM's analysis includes a comparison to budgeted results. Segment assets provided to the CODM are consistent with those reported on the Condensed Consolidated Balance Sheets with particular emphasis on the Company's available liquidity including cash, cash equivalents, investments, accounts receivable and inventory.

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

Three Months EndedMarch 31,
20262025
Total revenues$766,208$745,145
Less:
Cost of sales194,999151,558
R&D expenses
Research and early pipeline87,29190,470
Later-stage clinical programs36,17714,291
Marketed products55,32853,970
SG&A expenses
S&M expenses124,927102,531
G&A expenses133,363103,585
Other segment expense, net (1)28,59643,054
Net income$105,527$185,686

(1)Other segment expense, net during the three months ended March 31, 2026 and 2025 include intangible asset amortization, interest income and expense, other income (expense) and income tax expense.

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

Three Months EndedMarch 31,
20262025
VOXZOGO$219,880$213,754
Enzyme Therapies:
VIMIZIM210,204188,346
NAGLAZYME130,074114,290
PALYNZIQ89,55893,269
BRINEURA47,10440,362
ALDURAZYME36,74248,977
KUVAN23,88025,136
ROCTAVIAN2,63610,510
Total net product revenues760,078734,644
Royalty and other revenues6,13010,501
Total revenues$766,208$745,145

The Company considers there to be revenue concentration risks for regions where Net Product Revenues exceed 10% of consolidated Net Product Revenues. The concentration of the Company’s Net Product Revenues within the regions below may have a material adverse effect on the Company’s revenues and results of operations if sales in the respective regions experience difficulties. The table below disaggregates total Net Product Revenues by geographic region, which is based on patient location for the Company's commercial products sold directly by the Company, except for ALDURAZYME, which is distributed, marketed and sold exclusively by Sanofi worldwide.

Three Months EndedMarch 31,
20262025
United States$242,944$241,694
Europe218,836229,578
Latin America108,16488,282
Rest of world153,392126,113
Total net product revenues marketed by the Company723,336685,667
ALDURAZYME net product revenues marketed by Sanofi36,74248,977
Total net product revenues$760,078$734,644

The following table illustrates the percentage of the Company’s total Net Product Revenues attributed to the Company’s largest customers for the periods presented.

Three Months EndedMarch 31,
20262025
Customer A15%14%
Customer B1112
Customer C1011
Total36%37%

Concentration Information

On a consolidated basis, one customer accounted for 20% of the Company’s March 31, 2026 accounts receivable balance compared to December 31, 2025, when two customers accounted for 20% and 10% of the accounts receivable balance, respectively. As of March 31, 2026, and December 31, 2025, the accounts receivable balance for Sanofi included $152.6 million and $148.0 million, respectively, of unbilled accounts receivable, which becomes payable to the Company when the product is sold through by Sanofi. The Company does not require collateral from its customers, but does perform periodic credit evaluations of its customers’ financial condition and requires prepayments in certain circumstances.

The Company is mindful that conditions in the current macroeconomic environment, such as inflation, changes in interest and foreign currency exchange rates, natural disasters, geopolitical instability, wars and military conflicts, impact of new or increased tariffs and escalating trade tensions, regulatory uncertainty, and supply chain disruptions, could affect the Company’s ability to achieve its goals. In addition, the Company sells its products in countries that face economic volatility and weakness. Although the Company has historically collected receivables from customers in such countries, sustained weakness or further deterioration of the local economies and currencies may cause customers in those countries to delay payment or be unable to pay for the Company’s products. The Company believes that the allowances for doubtful accounts related to these countries, if any, are adequate as of March 31, 2026 based on its analysis of the specific business circumstances and expectations of collection for each of the underlying accounts in these countries. The Company will continue to monitor these conditions and will attempt to adjust its business processes, as appropriate, to mitigate macroeconomic risks to its business.

(9)STOCK-BASED COMPENSATION

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

Three Months EndedMarch 31,
20262025
Cost of sales$3,592$2,191
Research and development11,65412,127
Selling, general and administrative28,21223,382
Total stock-based compensation expense$43,458$37,700

(10)RESTRUCTURING

In connection with the Company’s plan to simplify its organizational design and strategic initiatives, the Company committed to plans in the first quarter of 2026 to reduce its global workforce. These reductions are expected to be substantially complete by the end of 2026. The reorganization plan and strategic initiatives comprised of severance charges of $8.3 million that were included in Selling, General and Administrative in the Condensed Consolidated Financial Statements of Comprehensive Income during the three months ended March 31, 2026.

The following unpaid balance as of March 31, 2026 was recorded to Accounts Payable and Accrued Liabilities on the Condensed Consolidated Balance Sheets:

Severance and related costs
Balance as of December 31, 2025$3,180
Severance charges8,312
Payments(205)
Balance as of March 31, 2026$11,287

(11)EARNINGS PER COMMON SHARE

Potentially issuable shares of common stock include shares issuable upon the exercise of outstanding employee stock option awards, common stock issuable under the ESPP, unvested RSUs and contingent issuances of common stock related to the Company’s convertible debt.

The following table sets forth the computation of basic and diluted earnings per common share (common shares in thousands):

Three Months EndedMarch 31,
20262025
Numerator:
Net income, basic$105,527$185,686
Add: Interest expense, net of tax, on the Company's convertible debt1,8341,833
Net income, diluted$107,361$187,519
Denominator:
Weighted-average common shares outstanding, basic192,497190,967
Effect of dilutive securities:
Common stock issuable under the Company's equity incentive plans8091,142
Common stock issuable under the Company’s convertible debt4,3654,365
Weighted-average common shares outstanding, diluted197,671196,474
Earnings per common share, basic$0.55$0.97
Earnings per common share, diluted$0.54$0.95

In addition to the equity instruments included in the table above, the table below presents potential shares of common stock that were excluded from the computation of diluted earnings per common share as they were anti-dilutive (in thousands):

Three Months EndedMarch 31,
20262025
Common stock issuable under the Company's equity incentive plans13,80311,686

(12)COMMITMENTS AND CONTINGENCIES

Contingencies

From time to time the Company is involved in legal actions arising in the normal course of its business. The process of resolving matters through litigation or other means is inherently uncertain and it is possible that an unfavorable resolution of these matters could adversely affect the Company, its results of operations, financial condition or cash flows. The Company’s general practice is to expense legal fees as services are rendered in connection with legal matters, and to accrue for liabilities when losses are probable and reasonably estimable based on existing information. The Company accrues for the best estimate of a loss within a range; however, if no estimate in the range is better than any other, then the minimum amount in the range is accrued. Liabilities are evaluated and refined each reporting period as additional information is known. Any receivables for insurance recoveries for these liability claims are recorded as assets when it is probable that a recovery will be realized.

As first disclosed in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023, the Company received a subpoena from the U.S. Department of Justice (DOJ) requesting that the Company produce certain documents regarding sponsored testing programs relating to VIMIZIM and NAGLAZYME. The Company has produced the requested documents in response to the subpoena and is cooperating fully. The Company is unable to make any assurances regarding the outcome of the investigation by the DOJ, or the impact, if any, that such investigation may have on the Company’s business and financial statements.

Other Commitments

The Company uses experts and laboratories at universities and other institutions to perform certain R&D activities. These amounts are recorded as R&D expense as services are provided. In the normal course of business, the Company enters into various firm purchase commitments primarily to procure active pharmaceutical ingredients, certain inventory-related items and certain third-party R&D services, production services and facility construction services. As of March 31, 2026, such commitments were estimated at $593.6 million, of which $327.8 million is expected to be paid in 2026 as underlying goods and services are received. The Company has also licensed technology from third parties, for which it is required to pay royalties upon future sales, subject to certain annual minimums.

(13)SUBSEQUENT EVENTS

On April 27, 2026, the Company completed its previously announced acquisition of Amicus, a publicly traded, global, biotechnology company for $14.50 per share in an all cash transaction for a total equity value of approximately $4.8 billion. In connection with the acquisition, the Company also repaid in full all outstanding loans, together with interest and all other amounts due in connection with such repayment, of Amicus for approximately $433.0 million. The acquisition is expected to strengthen the Company's commercial portfolio by 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 DMX-200, a potential first-in-class investigational small molecule for the treatment of focal segmental glomerulosclerosis (FSGS), a rare fatal kidney disease in Phase 3 development.

The transaction was financed through a combination of cash on hand and non-convertible debt financing. In connection with the acquisition, the Company obtained senior secured term loan facilities for $2.8 billion in aggregate principal and a new $600.0 million senior secured revolving credit facility. Borrowings under the term loan facilities and the 2026 revolving facility bear interest at the applicable interest rates specified in the governing credit agreement (the 2026 Credit Agreement). The 2026 Credit Agreement contains customary affirmative and negative covenants, including, among others, covenants that restrict the Company’s ability to incur additional indebtedness, create liens, make investments, pay dividends or make other restricted payments, dispose of assets, and enter into transactions with affiliates, in each case subject to exceptions set forth in the 2026 Credit Agreement. Upon entry into the senior secured term loan facility, the remaining Bridge Commitment was reduced from $2.8 billion tozero, and the Company recognized approximately $17.5 million of commitment fees that were deferred on the Condensed Consolidated Balance Sheets as of March 31, 2026.

The accounting impact of this acquisition and the results of operations for Amicus will be included in the Company’s Consolidated Financial Statements beginning in the second quarter of 2026. The initial accounting for this acquisition is incomplete, pending identification and measurement of the assets acquired and liabilities assumed.

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. Our San Rafael, California-based company, founded in 1997, has a proven track record of innovation, with a portfolio of commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, we seek to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients.

A summary of our commercial products, as of March 31, 2026, is provided below:

Commercial ProductsIndication
VOXZOGO (vosoritide)Achondroplasia
Enzyme Therapies:
VIMIZIM (elosulfase alpha)Mucopolysaccharidosis (MPS) IVA
NAGLAZYME (galsulfase)MPS VI
PALYNZIQ (pegvaliase-pqpz)Phenylketonuria (PKU)
BRINEURA (cerliponase alfa)Neuronal ceroid lipofuscinosis type 2 (CLN2)
ALDURAZYME (laronidase)MPS I
KUVAN (sapropterin dihydrochloride)PKU
ROCTAVIAN (valoctocogene roxaparvovec)(1)Severe Hemophilia A

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

Financial Highlights

Key components of our results of operations include the following:

Three Months EndedMarch 31,
20262025
Total revenues$766.2$745.1
Cost of sales$195.0$151.6
Research and development (R&D)$178.8$158.7
Selling, general and administrative (SG&A)$258.3$206.1
Provision for income taxes$35.7$52.4
Net income$105.5$185.7

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

Uncertainty Relating to Macroeconomic Environment

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

Recent Developments

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

In April 2026, we completed the acquisition of Amicus Therapeutics, Inc. (Amicus), a publicly traded, global, biotechnology company for $14.50 per share in an all-cash transaction for a total equity value of approximately $4.8 billion. In connection with the acquisition, the Company also repaid in full all outstanding loans, together with interest and all other amounts due in connection with such repayment, of Amicus for approximately $433.0 million. The acquisition was financed through a combination of cash on hand and non-convertible debt financing. The acquisition is expected to strengthen our commercial portfolio by adding two new treatments to our existing portfolio of medicines that target lysosomal storage diseases: GALAFOLD (migalastat), the first oral treatment for Fabry disease, and POMBILITI (cipaglucosidase alfa-atga) + OPFOLDA (miglustat), a two-component therapy for Pompe disease. In connection with the acquisition, we also now have U.S. rights to DMX-200, a potential first-in-class investigational small molecule for the treatment of focal segmental glomerulosclerosis (FSGS), a rare fatal kidney disease in Phase 3 development. The accounting impact of this acquisition and the results of operations for Amicus will be included in our Consolidated Financial Statements beginning in the second quarter of 2026. The initial accounting for this acquisition is incomplete, pending identification and measurement of the assets acquired and liabilities assumed. See Note 6 and Note 13 to our accompanying Condensed Consolidated Financial Statements for additional information regarding the acquisition.

In April 2026, in connection with the Amicus acquisition, we obtained senior secured term loan facilities for $2.8 billion in aggregate principal and a new $600.0 million senior secured revolving credit facility. Upon entry into the senior secured term loan facilities, the remaining bridge commitment was reduced from $2.8 billion to zero. In February 2026, we issued $850.0 million in aggregate principal amount of 5.5% senior unsecured notes due 2034 (the 2034 Notes), and the proceeds from the issuance were deposited into an escrow account as of March 31, 2026. The proceeds from the 2034 Notes and the secured term loan facilities were used to finance a portion of the Amicus acquisition that closed in April 2026. No amounts have been drawn under the senior secured revolving credit facility. See “Financial Condition, Liquidity and Capital Resources” below for additional information.

In April 2026, the first patient was enrolled in the registration-enabling Phase 2/3 study of BMN 333, our long-acting C-type natriuretic peptide (CNP) for achondroplasia.

In April 2026, we submitted its U.S. supplemental new drug application (sNDA) for full approval of VOXZOGO for achondroplasia.

In March 2026, we presented initial Phase 1/2 data for BMN 351 at the Muscular Dystrophy Association (MDA) Clinical & Scientific Congress demonstrating dose-dependent increases in dystrophin expression at Week 25 biopsy in both the 6 and 9 mg/kg dose cohorts. Clinical biomarkers, including decreases in creatine kinase, suggested improvements in overall muscle health beyond the Week 25 time point, and longer-term outcomes from both NSAA and 6MWT indicated a prevention of functional decline when compared to historical matched controls.

In March 2026, we announced our decision to discontinue dosing and enrollment in our Phase 2 trials for VOXZOGO in Turner Syndrome, SHOX-deficiency and Aggrecan (ACAN)-deficiency.

In February 2026, U.S. Food and Drug Administration (FDA) approved PALYNZIQ for adolescents 12 years of age and older with phenylketonuria (PKU).

See the risk factors described under “Business and Operational Risks” section in “Risk Factors” in Part II, Item 1A of this Quarterly Report on Form 10-Q.

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