16:02:26 EDT Tue 09 Jun 2026
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BioMarin Reports Fourth Quarter and Full-year 2025 Financial and Operating Results

2026-02-23 16:05 ET - News Release

BioMarin Reports Fourth Quarter and Full-year 2025 Financial and Operating Results

PR Newswire

Full-year 2025 Total Revenues Increased 13% Y/Y to $3.2 Billion, Led by 9% Revenue Growth for Enzyme Therapies and 26% Revenue Growth for VOXZOGO®

Fourth Quarter 2025 Total Revenues Increased 17% Y/Y Led by 13% Revenue Growth for Enzyme Therapies and 31% Revenue Growth for VOXZOGO

Announced Definitive Agreement to Acquire Amicus Therapeutics, including Galafold® for Fabry Disease and Pombiliti® + Opfolda® for Pompe Disease; Expected to Significantly Accelerate and Diversify Revenues

BioMarin Provides 2026 Guidance Excluding any Post-Close Contribution from the Announced Acquisition of Amicus, Anticipated to Close in Q2'26

Conference Call and Webcast Scheduled Today at 4:30 p.m. ET

SAN RAFAEL, Calif., Feb. 23, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced financial results for the fourth quarter and full year ended December 31, 2025.

"In 2025, operational excellence led to strengthening financial results, including double-digit topline growth, strong profitability and increasing cash flow. We also advanced multiple medicines in our pipeline and closed the year by announcing the acquisition of Amicus," said Alexander Hardy, President and Chief Executive Officer of BioMarin. "The Amicus transaction, which is expected to close in the second quarter, represents a compelling opportunity to reach more patients around the world and further strengthen our revenue growth through the next decade."

"We expect to build on this success in 2026, with another year of strong financial performance and momentum across the business. We look forward to adding Galafold and Pombiliti + Opfolda to our growing commercial enzyme therapies business, and to continued strong growth from VOXZOGO. Beyond our current commercial portfolio, we are excited by the progress we are seeing across our R&D pipeline and look forward to a multitude of pipeline catalysts throughout the year. These include three major data read-outs to support regulatory approvals, two age label expansions, plus the advancement of multiple clinical programs position us for significant portfolio progress. We are energized by what lies ahead this year and intend to deliver again on an ambitious set of priorities, demonstrating our dedication to innovation and sustained growth in ways that we believe will benefit patients, employees, and shareholders."

2025 Business Highlights

Innovation

  • Accelerated development of BMN 333, BioMarin's long-acting C-type natriuretic peptide (CNP), with Phase 1 PK data exceeding targeted free CNP exposure levels, reflecting its potential to become the new standard of care in achondroplasia.
  • Advanced five new VOXZOGO indications within the CANOPY program, including a pivotal Phase 3 study in hypochondroplasia and Phase 2 studies in idiopathic short stature, Noonan syndrome, Turner syndrome, and SHOX deficiency.
  • Reported positive data from the PALYNZIQ® Phase 3 PEGASUS study in 12- to 17-year-olds demonstrating statistically significant reductions in blood phenylalanine (Phe) compared to diet alone for adolescents with PKU.
  • Progressed BMN 351 for Duchenne muscular dystrophy, with initial Phase 1/2 data demonstrating 5.0% mean absolute dystrophin expression (without double-correction for histologic adjustment for muscle content) at week 25 in the 9 mg/kg cohort. The 12 mg/kg dose cohort continues to enroll participants, with topline data readout from this cohort expected in 2H'26.

Growth

  • Strong patient demand across the portfolio fueled 13% Y/Y full-year 2025 total revenue growth.
  • Enzyme Therapies full-year 2025 revenue advanced 9% Y/Y, supported by sustained high market penetration and patient adherence, led by robust 22% Y/Y growth from PALYNZIQ.
  • VOXZOGO generated 26% Y/Y revenue growth for full-year 2025, driven by deeper market penetration and increasing demand for the treatment of achondroplasia across 55 commercial markets. Markets outside of the U.S. (OUS) drove approximately 73% of VOXZOGO revenue in full-year 2025, reflecting the therapy's strong uptake across global markets.

Value Commitment

  • Announced the acquisition of Amicus Therapeutics in December 2025, expected to close in Q2'26, subject to regulatory clearances, approval by the stockholders of Amicus and other customary closing conditions. The addition of high-growth products, Galafold for Fabry Disease and Pombiliti + Opfolda for Pompe Disease, is expected to accelerate BioMarin's revenue growth and increase profitability. Adolescent label expansion for Pombiliti + Opfolda is anticipated in 2H'26.
  • Generated operating cash flows totaling $100 million in fourth quarter 2025 and $828 million for the full year. Total cash and investments totaled approximately $2 billion at year-end, and continued increasing operating cash flow is expected to support sustained investment in innovation and future growth.
  • Strong performance in 2025 led to significant GAAP and Non-GAAP Diluted Earnings per Share expansion, excluding acquired in-process research and development (IPR&D) charges related to the acquisition of Inozyme, $1.10 per share, and an inventory write-off related to ROCTAVIAN®, totaling $0.46 per share after tax.
  • The company secured financing of approximately $3.7 billion of non-convertible debt to support the Amicus acquisition with strong demand, achieving favorable pricing across the capital structure.

Anticipated 2026 Program Updates

VOXZOGO:

  • Phase 3 hypochondroplasia data 1H'26; regulatory submissions 2H'26
  • U.S. supplemental new drug application (sNDA) for full approval of VOXZOGO in achondroplasia Q2'26
  • Advancing Phase 2 studies in idiopathic short stature, Noonan syndrome, Turner syndrome, SHOX deficiency

BMN 333 (long-acting CNP):

  • Initiate registration-enabling Phase 2/3 study in achondroplasia 1H'26

PALYNZIQ:

  • U.S. PDUFA date for the adolescent label expansion February 28, 2026; EU approval 2026

BMN 401:

  • Phase 3 topline data in 1 to 12 year-old population with ENPP1 deficiency 1H'26; global regulatory submissions 2H'26; potential first?in?disease launch 2027

BMN 351

  • Phase 1/2 data presentation for 6 mg/kg and 9 mg/kg cohorts at Muscular Dystrophy Association (MDA) Clinical & Scientific Congress (March 8-11, 2026)

ROCTAVIAN

  • Following the company's October announcement to explore options to divest ROCTAVIAN, BioMarin undertook a comprehensive effort to identify a potential buyer. Despite these efforts, BioMarin was unable to identify a qualified buyer and has made the decision to voluntarily withdraw ROCTAVIAN from the market.

Fourth Quarter 2025 Financial Highlights

  • Total Revenues for the fourth quarter of 2025 were $875 million, an increase of 17% compared to the same period in 2024, driven by 31% year-over-year VOXZOGO revenue growth from new patients initiating therapy across all regions and the timing of large government orders, primarily in Latin America. In the quarter, revenues from BioMarin's Enzyme Therapies (ALDURAZYME®, BRINEURA®, NAGLAZYME®, PALYNZIQ and VIMIZIM®) also increased by 13% compared to the fourth quarter of 2024, driven by a combination of increased patient demand in all regions and the timing of large government orders.
  • GAAP Net Loss was $47 million for the fourth quarter of 2025 compared to GAAP Net Income of $125 million for the same period in 2024. The increase in GAAP Net Loss was primarily due to the company's strategic decision to voluntarily withdraw ROCTAVIAN from the market resulting in charges of approximately $240 million during the quarter. These charges were mainly comprised of $119 million of an inventory write-off that was included in Cost of Sales and $118 million of long-lived asset impairments included in Selling, General and Administrative expense. The increase in GAAP Net Loss was partially offset by improved revenue growth as mentioned above and lower provision for income taxes.
  • Non-GAAP Income for the fourth quarter of 2025 decreased to $89 million compared to $180 million for the same period in 2024. The decrease in Non-GAAP Income was primarily due to the ROCTAVIAN inventory write- off included in Cost of Sales. The decrease in Non-GAAP Income was partially offset by improved revenue growth as mentioned above.

                            
        
          Financial Highlights 
 (in millions of U.S. dollars, except per share data, unaudited)




                                                                                                                       Three Months Ended                    Twelve Months Ended

                                                                                                                           December 31,                          December 31,


                                                                                                2025            2024                      % Change     2025      2024            % Change





 
          Total Revenues                                                                     $875            $747                          17 %   $3,221    $2,854                13 %





 
          Net Product Revenues by Product:



 
          VOXZOGO                                                                            $273            $208                          31 %     $927      $735                26 %





 
          Enzyme Therapies:



 VIMIZIM                                                                                       $206            $191                           8 %     $792      $740                 7 %



 NAGLAZYME                                                                                      120             110                           9 %      485       480                 1 %



 PALYNZIQ                                                                                       125             100                          25 %      433       355                22 %



 ALDURAZYME                                                                                      49              39                          26 %      209       184                14 %



 BRINEURA                                                                                        49              48                           2 %      186       169                10 %



 
          Total Enzyme Therapies Revenue                                                     $549            $488                          13 %   $2,105    $1,928                 9 %





 
          KUVAN(R)                                                                            $23             $28                        (18) %     $100      $121              (17) %



 
          ROCTAVIAN                                                                           $13             $11                          18 %      $36       $26                38 %





 GAAP Net Income (Loss) (1)                                                                   $(47)           $125                       (138) %     $349      $427              (18) %



 Non-GAAP Income (1)(2)                                                                         $89            $180                        (51) %     $614      $686              (10) %



 GAAP Operating Margin % (1)(3)                                                             (5.1) %         21.6 %                                12.7 %   17.0 %



 Non-GAAP Operating Margin % (1)(2)(5)                                                       15.1 %         31.1 %                                23.3 %   28.6 %



 GAAP Diluted Earnings (Loss) per Share (EPS)(1)(4)                                         $(0.24)          $0.64                       (138) %    $1.80     $2.21              (19) %



 Non-GAAP Diluted EPS (1)(2)(5)                                                               $0.46           $0.92                        (50) %    $3.15     $3.52              (11) %




 (1) 
 Includes acquired IPR&D charges of $221 million (or approximately $1.10 on a per share basis) related to acquisition of Inozyme for the twelve months ended December 31, 2025.



 (2)   Refer to Non-GAAP Information beginning on page 10 of this press release for definitions of Non-GAAP Income, Non-GAAP Operating Margin percentage and Non-GAAP Diluted EPS along with
          the related reconciliations to the comparable information reported under U.S. GAAP.




 (3) 
 GAAP Operating Margin percentage is defined by the company as GAAP Income (Loss) from Operations divided by Total Revenues.



 (4)   Includes approximately $240 million of restructuring charges (or approximately $0.94 after tax on a per share basis) related to the company's strategic decision to voluntarily withdraw
          ROCTAVIAN from the market for the three and twelve months ended December 31, 2025.



 (5)   Includes $119 million inventory write-off (or approximately $0.46 after tax on a per share basis) related to the company's strategic decision to voluntarily withdraw ROCTAVIAN from the
          market for the three and twelve months ended December 31, 2025.

Forward-Looking Non-GAAP Financial Information

BioMarin does not provide guidance for GAAP reported financial measures (other than revenue) or a reconciliation of forward-looking Non-GAAP financial measures to the most directly comparable GAAP reported financial measures because the company is unable to predict with reasonable certainty the financial impact of changes resulting from its strategic portfolio and business operating model reviews; potential future asset impairments; gains and losses on investments; and other unusual gains and losses without unreasonable effort. These items are uncertain, depend on various factors, and could have a material impact on GAAP reported results for the guidance period. As such, any reconciliations provided would imply a degree of precision that could be confusing or misleading to investors.

2026 Full-Year Financial Guidance (in millions, except EPS amounts)

  • 2026 guidance excludes any post-close contribution from the announced acquisition of Amicus Therapeutics, anticipated to close in Q2'26
  • Total Revenues guidance reflects expectation of continued strong patient demand across Enzyme Therapies and VOXZOGO in 2026
  • Other Revenue guidance reflects KUVAN, royalty revenue (including conclusion of U.S. Firdapse royalty term in January 2026), and the company's strategic decision to voluntarily withdraw ROCTAVIAN from the market
  • Non-GAAP Diluted EPS guidance includes approximately $0.25 of pre-close operating and interest expenses associated with the Amicus transaction
  • In 2026, excluding the impact of the Amicus transaction, Non-GAAP Operating Margin is expected to be approximately 40% for the full year

 
            Item           2025 Actuals        2026 Guidance



 Total Revenues                    $3,221 $3,325   to       $3,425



 Enzyme Therapies                  $2,105 $2,225   to       $2,275



 VOXZOGO                             $927   $975   to       $1,025



 Other Revenues(1)                   $189   $100   to         $125



 Non-GAAP Diluted EPS (2)(3)        $3.15  $4.95   to        $5.15




 (1) 
 Other Revenues includes KUVAN, ROCTAVIAN, and royalties



 (2)   Refer to Non-GAAP Information beginning on page 10 of this press release for definition of
          Non-GAAP Diluted EPS.




 (3)   Non-GAAP Diluted EPS guidance assumes approximately 200 million Weighted-Average Diluted
          Shares Outstanding.

BioMarin will host a conference call and webcast to discuss fourth quarter 2025 financial results today, Monday, February 23, 2026, at 4:30 p.m. ET. This event can be accessed through this link or on the investor section of the BioMarin website at www.biomarin.com.


 U.S./Canada Dial-in Number: 800-715-9871      Replay Dial-in Number: 800-770-2030



 International Dial-in Number:  646-307-1963   Replay International Dial-in Number:
                                                 609-800-9909



 Conference ID:  4503000                     
 Conference ID: 4503000

About BioMarin

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 multiple 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. To learn more, please visit www.biomarin.com.

Forward-Looking Statements

This press release and the associated conference call and webcast contain forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: future financial performance, including the expectations of Total Revenues, Non-GAAP Operating Margin percentage, and Non-GAAP Diluted EPS for the full-year 2026 and future periods, and the underlying drivers of those results, such as the expected demand and continued growth of BioMarin's Enzyme Therapies portfolio, the expected growth from VOXZOGO, and the expected impact of Other Revenues; the anticipated closing and benefits of BioMarin's proposed acquisition of Amicus Therapeutics, Inc.; BioMarin's plans for investment in innovation and future growth; the timing of orders for commercial products; plans and expectations regarding the development, commercialization and commercial prospects of BioMarin's product candidates and commercial products, including the prospects and timing of actions relating to clinical studies and trials and product approvals, such as study initiations, study advancements, data readouts, submissions, filings, approvals, and label expansions; the expected benefits and availability of BioMarin's commercial products and product candidates; and potential growth opportunities and trends, including the assumptions and expectations regarding total addressable patient population (TAPP) with respect to the conditions targeted by BioMarin's product candidates and commercial products.

These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: BioMarin's success in the commercialization of its commercial products; BioMarin's ability to consummate and realize the anticipated benefits of any acquisitions; impacts of macroeconomic and other external factors on BioMarin's operations, regulatory uncertainty, the impact of new or increased tariffs, other trade protection measures, and escalating trade tensions; results and timing of current and planned preclinical studies and clinical trials and the release of data from those trials; BioMarin's ability to successfully manufacture its commercial products and product candidates; the content and timing of decisions by the U.S. Food and Drug Administration, the European Medicines Agency, the European Commission and other regulatory authorities concerning each of the described products and product candidates; the market for each of these products; BioMarin's ability to meet product demand; actual sales of BioMarin's commercial products; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended September 30, 2025, as such factors may be updated by any subsequent reports. Investors are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

BioMarin®, VOXZOGO®, VIMIZIM®, NAGLAZYME®, PALYNZIQ®, BRINEURA®, KUVAN® and ROCTAVIAN® are registered trademarks of BioMarin Pharmaceutical Inc., or its affiliates. ALDURAZYME® is a registered trademark of BioMarin/Genzyme LLC. All other brand names and service marks, trademarks and other trade names appearing in this release are the property of their respective owners.

                                                      
      
            BIOMARIN PHARMACEUTICAL INC.

                                              
         
         CONDENSED CONSOLIDATED STATEMENTS OF INCOME

                                        
          
        Three and Twelve Months Ended December 31, 2025 and 2024

                                        
          
        (In thousands of U.S. dollars, except per share amounts)

                                                          
          
            (Unaudited)




                                                                                                                        Three Months Ended                  Twelve Months Ended

                                                                                                                        December 31,                  December 31,


                                                                                                                   2025             2024         2025                  2024





 
            REVENUES:



 Net product revenues                                                                                         $859,321         $735,634   $3,167,759            $2,809,445



 Royalty and other revenues                                                                                     15,244           11,679       53,494                44,470



 Total revenues                                                                                                874,565          747,313    3,221,253             2,853,915



 
            OPERATING EXPENSES:



 Cost of sales                                                                                                 275,709          136,139      717,442               580,235



 Research and development                                                                                      192,413          173,509      921,930               747,184



 Selling, general and administrative                                                                           446,207          266,607    1,153,017             1,009,025



 Intangible asset amortization                                                                                   4,846            9,651       19,386                43,257



 Gain on sale of nonfinancial assets                                                                                 -                                         (10,000)



 Total operating expenses                                                                                      919,175          585,906    2,811,775             2,369,701



 
            INCOME (LOSS) FROM OPERATIONS                                                                   (44,610)         161,407      409,478               484,214





 Interest income                                                                                                19,210           17,680       74,904                74,883



 Interest expense                                                                                              (2,778)         (2,577)    (10,899)             (12,666)



 Other income (expense), net                                                                                     1,025          (6,871)       8,997               (4,668)



 
            INCOME (LOSS) BEFORE INCOME TAXES                                                               (27,153)         169,639      482,480               541,763



 Provision for income taxes                                                                                     19,420           44,696      133,579               114,904



 
            NET INCOME (LOSS)                                                                              $(46,573)        $124,943     $348,901              $426,859



 
            EARNINGS (LOSS) PER SHARE, BASIC                                                                 $(0.24)           $0.66        $1.82                 $2.25



 
            EARNINGS (LOSS) PER SHARE, DILUTED                                                               $(0.24)           $0.64        $1.80                 $2.21



 Weighted average common shares outstanding, basic                                                             192,225          190,688      191,787               190,027



 Weighted average common shares outstanding, diluted                                                           192,225          196,581      197,394               196,708

                                                                                    
          
            BIOMARIN PHARMACEUTICAL INC.

                                                                               
          
            CONDENSED CONSOLIDATED BALANCE SHEETS

                                                                              
          
            December 31, 2025
             and 2024

                                                                      
          
            (In thousands of U.S. dollars, except per share amounts)

                                                                                            
          
            (Unaudited)




                                                                                                                                                       December 31, 2025 December 31, 2024


                                                                                             
          
            ASSETS



          Current assets:



          Cash and cash equivalents                                                                                                                          $1,311,679           $942,842



          Short-term investments                                                                                                                                248,930            194,864



          Accounts receivable, net                                                                                                                              908,214            660,535



          Inventory                                                                                                                                           1,298,883          1,232,653



          Other current assets                                                                                                                                  185,784            201,533



          Total current assets                                                                                                                                3,953,490          3,232,427



          Noncurrent assets:



          Long-term investments                                                                                                                                 492,242            521,238



          Property, plant and equipment, net                                                                                                                    952,508          1,043,041



          Intangible assets, net                                                                                                                                213,837            255,278



          Goodwill                                                                                                                                              196,199            196,199



          Deferred tax assets                                                                                                                                 1,508,697          1,489,366



          Other assets                                                                                                                                          277,049            251,391



          Total assets                                                                                                                                       $7,594,022         $6,988,940


                                                                              
          
            LIABILITIES AND STOCKHOLDERS' EQUITY



          Current liabilities:



          Accounts payable and accrued liabilities                                                                                                             $759,031           $606,988



          Total current liabilities                                                                                                                             759,031            606,988



          Noncurrent liabilities:



          Long-term convertible debt, net                                                                                                                       597,176            595,138



          Other long-term liabilities                                                                                                                           150,816            128,824



          Total liabilities                                                                                                                                   1,507,023          1,330,950



          Stockholders' equity:



          Common stock, $0.001 par value: 500,000,000 shares authorized; 192,300,091 and                                                                            192                191
190,761,349 shares issued and outstanding, respectively



          Additional paid-in capital                                                                                                                          5,956,582          5,802,068



          Company common stock held by the Nonqualified Deferred Compensation Plan                                                                             (10,508)          (11,227)



          Accumulated other comprehensive income (loss)                                                                                                        (13,473)            61,653



          Retained earnings (accumulated deficit)                                                                                                               154,206          (194,695)



          Total stockholders' equity                                                                                                                          6,086,999          5,657,990



          Total liabilities and stockholders' equity                                                                                                         $7,594,022         $6,988,940



                                                          
          
            BIOMARIN PHARMACEUTICAL INC.

                                                
          
            CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS

                                          
          
            Twelve Months Ended December 31, 2025
             and 2024

                                                         
          
            (In thousands of U.S. dollars)

                                                                  
          
            (Unaudited)




                                                                                                                                         Twelve Months Ended December 31,


                                                                                                                                    2025               2024



 
            CASH FLOWS FROM OPERATING ACTIVITIES:



 Net income                                                                                                                    $348,901           $426,859



 Adjustments to reconcile net income to net cash provided by operating activities:



 Depreciation and amortization                                                                                                   79,557             96,426



 Non-cash interest expense                                                                                                        2,622              3,359



 Accretion of discount on investments                                                                                           (4,801)           (8,345)



 Stock-based compensation                                                                                                       181,409            201,571



 Gain on sale of nonfinancial assets                                                                                                  -          (10,000)



 Impairment of assets                                                                                                           125,012             19,889



 ROCTAVIAN inventory write-off                                                                                                  119,208



 Deferred income taxes                                                                                                           48,738             56,096



 Unrealized foreign exchange gain                                                                                                 4,459           (16,753)



 Acquired in-process research & development expense                                                                             220,963



 Other                                                                                                                          (4,414)            20,135



 Changes in operating assets and liabilities:



 Accounts receivable, net                                                                                                     (228,054)          (57,909)



 Inventory                                                                                                                    (116,929)          (63,530)



 Other current assets                                                                                                             8,891            (3,778)



 Other assets                                                                                                                  (38,573)          (73,700)



 Accounts payable and accrued liabilities                                                                                        66,136           (32,240)



 Other long-term liabilities                                                                                                     14,869             14,761



 Net cash provided by operating activities                                                                                      827,994            572,841



 
            CASH FLOWS FROM INVESTING ACTIVITIES:



 Purchases of property, plant and equipment                                                                                   (103,038)          (85,424)



 Maturities and sales of investments                                                                                            337,801            633,018



 Purchases of investments                                                                                                     (355,875)         (410,250)



 Proceeds from sale of nonfinancial assets                                                                                            -            10,000



 Purchase of intangible assets                                                                                                  (7,937)          (11,994)



 Acquisition, net of cash acquired                                                                                            (285,193)



 Other                                                                                                                                -             1,141



 Net cash provided by (used in) investing activities                                                                          (414,242)           136,491



 
            CASH FLOWS FROM FINANCING ACTIVITIES:



 Proceeds from exercises of awards under equity incentive plans                                                                  14,460             49,277



 Taxes paid related to net share settlement of equity awards                                                                   (55,965)          (77,560)



 Repayments of convertible debt                                                                                                       -         (494,987)



 Other                                                                                                                            (889)           (3,177)



 Net cash used in financing activities                                                                                         (42,394)         (526,447)



 Effect of exchange rate changes on cash                                                                                        (2,521)             4,830



 
            NET INCREASE (DECREASE) IN CASH AND CASH EQUIVALENTS                                                              368,837            187,715



 Cash and cash equivalents:



 Beginning of period                                                                                                           $942,842           $755,127



 End of period                                                                                                               $1,311,679           $942,842

Non-GAAP Information

The results presented in this press release include both GAAP information and Non-GAAP information. Non-GAAP Income is defined by the company as GAAP Net Income (Loss) excluding amortization of intangible assets, stock-based compensation expense and, in certain periods, certain other specified items, as detailed below when applicable. The company also includes a Non-GAAP adjustment for the estimated tax impact of the reconciling items. Non-GAAP R&D expenses and Non-GAAP SG&A expenses are defined by the company as GAAP R&D expenses and GAAP SG&A expenses, respectively, excluding stock-based compensation expense and, in certain periods, certain other specified items, as detailed below when applicable. Non-GAAP Operating Margin percentage is defined by the company as GAAP Income (Loss) from Operations, excluding amortization of intangible assets, stock-based compensation expense and, in certain periods, certain other specified items, divided by GAAP Total Revenues. Non-GAAP Diluted EPS is defined by the company as Non-GAAP Income divided by Non-GAAP Weighted-Average Diluted Shares Outstanding. Non-GAAP Weighted-Average Diluted Shares Outstanding is defined by the company as GAAP Weighted-Average Diluted Shares Outstanding, adjusted to include any common shares issuable under the company's equity plans or convertible debt in periods when they are dilutive under Non-GAAP.

BioMarin regularly uses both GAAP and Non-GAAP results and expectations internally to assess its financial operating performance and evaluate key business decisions related to its principal business activities: the discovery, development, manufacture, marketing and sale of innovative biologic therapies. BioMarin also uses Non-GAAP Income internally to understand, manage and evaluate its business and to make operating decisions, and compensation of executives is based in part on this measure. Because these Non-GAAP metrics are important internal measurements for BioMarin, the company believes that providing this information in conjunction with BioMarin's GAAP information enhances investors' and analysts' ability to meaningfully compare the company's results from period to period and to its forward-looking guidance, and to identify operating trends in the company's principal business.

Non-GAAP financial measures are not meant to be considered in isolation or as a substitute for, or superior to comparable GAAP measures and should be read in conjunction with the consolidated financial information prepared in accordance with GAAP. Investors should note that the Non-GAAP information is not prepared under any comprehensive set of accounting rules or principles and does not reflect all of the amounts associated with the company's results of operations as determined in accordance with GAAP. Investors should also note that these Non-GAAP financial measures have no standardized meaning prescribed by GAAP and, therefore, have limits in their usefulness to investors. In addition, from time to time in the future there may be other items that the company may exclude for purposes of its Non-GAAP financial measures; likewise, the company may in the future cease to exclude items that it has historically excluded for purposes of its Non-GAAP financial measures. Because of the non-standardized definitions, the Non-GAAP financial measure as used by BioMarin in this press release and the accompanying tables may be calculated differently from, and therefore may not be directly comparable to, similarly titled measures used by other companies.

The following tables present the reconciliation of GAAP reported to Non-GAAP adjusted financial information:

                     
          
            Reconciliation of GAAP Reported Information to Non-GAAP Information (1)

                               
          
            (In millions of U.S. dollars, except per share data)

                                                   
          
            (unaudited)




                                                                                                                     Three Months Ended                Twelve Months Ended

                                                                                                                     December 31,                December 31,


                                                                                                         2025            2024             2025    2024





 
            GAAP Reported Net Income (Loss)                                                          $(47)           $125             $349    $427



 Adjustments



 Stock-based compensation expense - COS                                                                    4               3               14      15



 Stock-based compensation expense - R&D                                                                   14              14               55      60



 Stock-based compensation expense - SG&A                                                                  29              34              113     127



 Amortization of intangible assets                                                                         5              10               19      43



 Acquisition-related costs (2)                                                                             -                             15



 Gain on sale of nonfinancial assets (3)                                                                   -                                  (10)



 Severance and restructuring costs (4)                                                                   124              10              124      96



 Loss on investments (5)                                                                                   -                              3       5



 Income tax effect of adjustments                                                                       (40)           (16)            (78)   (76)



 Non-GAAP Income                                                                                         $89            $180             $614    $686

                                           
   
           Three Months Ended                        
     
   Twelve Months Ended

                                             
   
            December 31,                               
   
     December 31,


                                                   2025                              2024                       2025                         2024


                                       R&D            SG&A                     R&D        SG&A     R&D                 SG&A         R&D            SG&A





          
            GAAP expenses $192             $446                     $174         $267     $922                $1,153        $747            $1,009



          Adjustments



          Stock-based                (14)            (29)                    (14)        (34)    (55)                (113)        (60)           (127)
compensation
expense



          Acquisition-related                                                                                         (15)
costs (2)



          Severance and                             (124)                                (10)                        (124)                        (96)
restructuring costs (4)



          Non-GAAP expenses          $178             $292                     $159         $222     $867                  $901        $688              $786

                                                                         Three Months Ended                                    Twelve Months Ended

                                                         
      
           December 31,                             
     
      December 31,


                                                   2025          Percent             2024           Percent   2025              Percent              2024              Percent
                                                        of GAAP                             of GAAP                    of GAAP                               of GAAP
                                                          Total                               Total                      Total                                 Total
                                                        Revenue                             Revenue                    Revenue                               Revenue





 
            GAAP Income (Loss) from Operations $(45)         (5.1) %             $161            21.6 %   $409               12.7 %              $484               17.0 %



 Adjustments



 Stock-based compensation expense                   47              5.4                51               6.8     182                  5.7                202                  7.1



 Amortization of intangible assets                   5              0.6                10               1.3      19                  0.6                 43                  1.5



 Acquisition-related costs (2)                                                                               15                  0.5



 Gain on sale of nonfinancial assets (3)                                                                                                         (10)               (0.4)



 Severance and restructuring costs (4)             124             14.2                10               1.3     124                  3.8                 96                  3.4



 Non-GAAP Income from Operations                  $132           15.1 %             $232            31.1 %   $750               23.3 %              $815               28.6 %

                                                      Three Months Ended                   Twelve Months Ended

                                                         December 31,                          December 31,


                                                 2025                        2024          2025                        2024





 
            GAAP Diluted EPS               $(0.24)                      $0.64         $1.80                       $2.21



 Adjustments



 Stock-based compensation expense              $0.24                       $0.26         $0.92                       $1.03



 Amortization of intangible assets             $0.03                       $0.05         $0.10                       $0.22



 Acquisition-related costs (2)           
 $       -      
          $        -        $0.08        
         $        -



 Gain on sale of nonfinancial assets (3) 
 $       -      
          $        -  
 $       -                    $(0.05)



 Severance and restructuring costs (4)         $0.63                       $0.05         $0.63                       $0.49



 Loss on investments (5)                 
 $       -      
          $        -        $0.02                       $0.03



 Income tax effect of adjustments            $(0.20)                    $(0.08)      $(0.40)                    $(0.39)



 Non-GAAP Diluted EPS                          $0.46                       $0.92       $3.15                       $3.52




 (1) 
 Certain amounts may not sum or recalculate due to rounding.



 (2) 
 These amounts were included in SG&A and represent severance costs incurred in the acquisition of Inozyme in July 2025.



 (3) 
 Represents a payment triggered by a third party's attainment of a regulatory approval milestone related to previously sold intangible assets.



 (4)   These amounts were included in SG&A and represent impairment of long-lived assets, severance and other restructuring costs related to the company's 2025 strategic decision to
          voluntarily withdraw ROCTAVIAN from the market and 2024 corporate initiatives and the associated organizational redesign efforts.



 (5) 
 Represents impairment loss on non-marketable equity securities recorded in Other income (expense), net.

                                                                           Three Months Ended             Twelve Months Ended

                                                                           December 31,             December 31,


                                                                 2025  2024            2025    2024





 
            GAAP Weighted-Average Diluted Shares Outstanding 192.2 196.6           197.4   196.7



 Adjustments



 Common stock issuable under the company's equity plans (1)      0.8



 Common stock issuable under the Company's convertible debt(1)   4.4



 Non-GAAP Weighted-Average Diluted Shares Outstanding          197.4 196.6           197.4   196.7




 (1) Common stock issuable under the company's equity plans and convertible debt were excluded from the computation of GAAP Weighted-Average Diluted Shares Outstanding for the three months
        ended December 31, 2025, as they were anti-dilutive.


 
 Contact:



 
 Investors:                   Media:



 
 Traci McCarty                Marni Kottle



 
 BioMarin Pharmaceutical Inc. BioMarin Pharmaceutical
                                  Inc.



 
 (415) 455-7558               (650) 374-2803

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SOURCE BioMarin Pharmaceutical Inc.

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