Slides
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Fourth Quarter and Full Year 2025 Earnings February 23, 2026
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2 Forward-Looking Statements This presentation 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, those factors detailed in BioMarin’s press release issued on February 23, 2026, and 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. Non-GAAP Financial Measures This presentation includes 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. Because such Non-GAAP metrics are important internal measurements for BioMarin, BioMarin believes that providing this information in conjunction with 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 presentation may be calculated differently from, and therefore may not be directly comparable to, similarly titled measures used by other companies. 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. With respect to historical Non-GAAP adjusted financial information, see the appendix beginning on slide 18 for the reconciliations to the comparable information reported under U.S. GAAP.
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Q4’25 and FY’25 Earnings Agenda: Key Business Updates Financial Results Commercial Update Research & Development Update Q&A 1 2 3 4 5 Alexander Hardy Chief Executive Officer Brian Mueller Chief Financial Officer Cristin Hubbard Chief Commercial Officer Greg Friberg Chief R&D Officer
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4 Q4’25 and FY’25 Key Business Updates Alexander Hardy Chief Executive Officer
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5 Record 2025 Performance Builds Strong Momentum into 2026 GROWTH 51 53 488 549 208 273 Q4 2024 Q4 2025 Enzyme TherapiesVOXZOGO Other Revenues $747 $875 Total Revenues +17% Q4 Y/Y +13% FY Y/Y 191 189 1,928 2,105 735 927 FY 2024 FY 2025 $ in millions $2,854 $3,221 Anticipated Key Highlights in 20261 Expand Reach with New Assets ENPP1 deficiency BMN 401 Fabry Disease Pompe Disease 1Pending closure of BioMarin’s acquisition of Amicus Therapeutics (including marketed products Galafold and Pombiliti + Opfolda); acquisition is expected to close in Q2’26, subject to regulatory clearances, approval by the stockholders of Amicus and other customary closing conditions Advance Lifecycle Innovation Deliver Strong Results Across Portfolio hypochondroplasia BMN 333 achondroplasia PKU adolescents
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6 Q4’25 and FY’25 Financial Results Brian Mueller Chief Financial Officer
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7 Q4 and FY’25 Growth Driven by Robust Global Demand across Portfolio 51 53 488 549 208 273 Q4 2024 Q4 2025 Enzyme TherapiesVOXZOGO Other Revenues $747 Total Revenues $875 Q4 +31% +13% VOXZOGO • Sustained strong global expansion; FY’25 revenue ~73% outside of the U.S. (OUS) • Patient additions across all regions • Q4’25 benefited from large OUS contracted order (not expected to repeat in Q1’26) Enzyme Therapies • FY’25 revenue increased 9%, Y/Y • PALYNZIQ: Fourth consecutive quarter of 20% or higher Y/Y revenue growth+4% FY +26% +9% (1%) Y/Y Change +17% +13% $ in millions
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8 FY’25 Results Reflect Strong Execution and Underlying Performance GROWTH FY 2025 Results In millions, except percentages and per share amounts FY’25 Y/Y Change GAAP R&D $922 +23% GAAP SG&A $1,153 +14% GAAP Operating Margin 12.7% (4.3) ppts Non-GAAP R&D1 $867 +26% Non-GAAP SG&A1 $901 +15% Non-GAAP Operating Margin1 23.3% (5.3) ppts GAAP Diluted EPS $1.80 (19%) Non-GAAP Diluted EPS1 $3.15 (11%) Operating Cash Flow $828 +45% FY’25 Non-GAAP Diluted EPS • $3.15 EPS includes ($0.46) from ROCTAVIAN inventory write-off and ($1.10) acquired IPR&D from Inozyme acquisition • Strong execution drove underlying profitability expansion FY’25 Operating Cash Flow • Significant operating cash flow growth continues, with $828M generated in FY’25, providing capital to reinvest in innovation and future growth 1Refer to slide 2 for more detail on Non-GAAP financial measures ROCTAVIAN Charges Incurred in Q4’25 • $240M ($0.94 after-tax diluted EPS impact) charges on a GAAP basis due to the company’s decision to voluntarily withdraw ROCTAVIAN from the market • $119M ($0.46 after-tax diluted EPS impact) inventory write-off reflected in GAAP and Non-GAAP results
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9 Full-year 2026 Guidance (Excludes Post-close Contributions from Amicus Acquisition1) (In millions, except per share amounts) 2025 Actual 2026 Guidance As of February 23, 2026 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 $189 $100 to $125 Non-GAAP Diluted EPS2,3 $3.15 $4.95 to $5.15 1Post-close contributions refer to revenue, operating expense and incremental interest expense associated with acquisition financing after closing of the Amicus transaction; 2Refer to slide 2 for more detail on Non-GAAP financial measures; 3Non-GAAP Diluted EPS guidance assumes approximately 200 million Weighted-Average Diluted Shares Outstanding. Key Considerations • 2026 guidance expected to be updated upon the closing of the Amicus acquisition, anticipated 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 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 2026 Anticipated Quarterly Dynamics • Due to order timing, Q1’26 Total Revenues and VOXZOGO revenue expected to be similar to Q1’25 • Q1’26 Non-GAAP Diluted EPS expected to be lowest of 2026 due to revenue weighting and pre- close Amicus costs • Enzyme Therapies and VOXZOGO: Large OUS orders to drive higher 2H revenue vs. 1H and weighted to Q4
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10 Commercial Update Cristin Hubbard Chief Commercial Officer
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11 Strong Enzyme Therapies Growth Led by PALYNZIQ GROWTH $433 $792 $485 $209 $186 PALYNZIQ VIMIZIM NAGLAZYME ALDURAZYME BRINEURA Growth across every product in Enzyme Therapies portfolio, Y/Y • 9% Y/Y growth driven by new patient starts across all products and consistently high adherence rates • Potential PALYNZIQ Label Expansion in 2026: • Only therapy to enable physiologic Phe levels while reducing dietary restrictions, regardless of severity • February 28, 2026: U.S. PDUFA target action date for anticipated adolescent label expansion • Caregiver support during titration period with PALYNZIQ may drive adherence during adulthood • Potential for PALYNZIQ-treated adolescents to benefit from Phe lowering and reduced need for dietary therapy +7% +1% +14% +10% Enzyme Therapies FY’25 Revenue +22% +9% Y/Y Enzyme Therapies $ in millions
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12 VOXZOGO: FY’25 26% Y/Y Driven by Global Expansion Skeletal Conditions Revenue Over 5,000 children with achondroplasia now treated with VOXZOGO; Global expansion expected in 2026 • Rapid uptake in newly launched markets in 2025 • In Q4’25, approximately half of new U.S. starts in under- 2-year age group • 2026 focused, multi-pronged global expansion: • Driving new starts across all ages worldwide, with emphasis on under-2-year age group • Continue to find and start treatment-eligible infants in highly penetrated incident markets • More deeply penetrate larger countries • Expand uptake in fast-growing, newly launched markets • sNDA submission for full approval of VOXZOGO expected Q2’26, leveraging extensive safety and efficacy data package $273 $927 Q4 2025 FY 2025 +31% +26% Percentages reflect year-over-year change $ in millions
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Research & Development Update Greg Friberg Chief R&D Officer 13
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14 Evidence that Cumulative Growth with VOXZOGO Treatment from Infancy Provides Clinically Important Health Benefits Neurological Foramen magnum stenosis & sleep testing • In a retrospective study, no children who initiated vosoritide aged <2 years developed foramen magnum stenosis and vosoritide initiation in children aged <3 years improved sleep quality1 Spinal Symptomatic spinal stenosis • In a prospective observational study, vosoritide improved spinal alignment in young children with ACH after 1 year of treatment2 Musculoskeletal Body proportionality • In clinical trials, vosoritide improved upper-to-lower body segment ratio vs untreated children3,4 Genu varum • In a prospective observational study, vosoritide improved genu varum in young children with ACH after 1 year of treatment2 Preventing complications Increased Functionality Improved mobility/gait • In an observational study, vosoritide provided clinically significant improvements in the 6-minute walking distance test from baseline after 1 year of treatment5 Improved QOL • In a clinical trial, vosoritide positively impacted physical and social QOL after early treatment initiation in children with ACH after 3 years6 Source: Savarirayan R, et al., ESPE/ESE 2025 oral presentation. 1Lourenco C, et al. 2025. Abstract at ACMG. P156 March 18–22, 2025. 2Sawamura K, et al. J Pediatr Orthop. 2025. doi:10.1097/BPO.0000000000002980. 3Savarirayan R, et al. Med. 2024. doi:10.1016/j.medj.2024.11.019. 4Savarirayan R, et al. Lancet Child Adolesc Health. 2024. doi:10.1016. 5Reincke S, et al. J Endo Societ. 2025. doi:10.1210. 6Savarirayan R, et al. Genet Med. 2024;26:101274. ACH, achondroplasia; QOL, quality of life. As presented at ESPE/ESE 2025
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15 BMN 333: Phase 2/3 Study Design for BioMarin’s Long-Acting CNP Registration-enabling Study Designed to Target Superior Profile; Planned for 1H’26 Start • Phase 2 dose-ranging portion planned to include three dose levels of BMN 333 vs. VOXZOGO • Phase 3 design: • Comparison of BMN 333 weekly against active control (VOXZOGO): 60 patients in each arm • 90% power to detect a 50% increase in annualized growth velocity (AGV) vs. VOXZOGO; infers a minimal detectable increase of 2.25 cm/yr over placebo • Will assess additional measures of health and wellness, including proportionality and quality of life • Goal is to establish BMN 333 as new standard of care for achondroplasia Key Highlights BMN 333 Phase 2/3 Study Design1 Enrollment to Begin 1H’26 BMN 333 350 µg/kg BMN 333 250 µg/kg BMN 333 150 µg/kg vosoritide Weight-band Dosing BMN 333 Selected Dose vosoritide Weight-band Dosing 1Study design subject to change post-feedback from regulatory authorities
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16 Anticipated 2026 Pipeline Highlights Across Therapeutic Areas 2 Label Expansions Expected for Adolescents1 PKU Pompe disease 2 Program Updates to Enable Registration-enabling Studies BMN 351 Duchenne muscular dystrophy (Phase 1/2 data) BMN 333 Long-acting CNP for achondroplasia (Phase 1 data) 6 New Opportunities across Skeletal Conditions 5 new indications: hypochondroplasia, idiopathic short stature, Noonan syndrome, Turner syndrome, SHOX deficiency BMN 333 Long-acting CNP 3 Major Data Readouts and Planned Submissions for Regulatory Approval hypochondroplasia (Phase 3 data) ENPP1 deficiency (Phase 3 data) 16 New product for achondroplasia achondroplasia Full Approval BMN 401 1Pending closure of BioMarin’s acquisition of Amicus (including Pombiliti + Opfolda); acquisition is expected to close in Q2’26, subject to regulatory clearances, approval by the stockholders of Amicus and other customary closing conditions
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17 Q&A
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Appendix Reconciliation of GAAP Reported to Selected Non-GAAP Adjusted Information
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19 Reconciliation of GAAP Reported Net Income to Non-GAAP Income(1) Please refer to slide 22 for footnotes to the table
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20 Reconciliation of GAAP and Non-GAAP R&D and SG&A Expenses(1) Please refer to slide 22 for footnotes to the table
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21 Reconciliation of GAAP Income from Operations to Non-GAAP Income from Operations(1) Please refer to slide 22 for footnotes to the table
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22 Reconciliation of GAAP Diluted EPS to Non-GAAP Diluted EPS(1) (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.