Slides
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1 Third Quarter 2026 Financial Results November 2, 2026
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2 Forward-Looking Statements Non-GAAP Financial Measures 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 Revenues and Non-GAAP Diluted EPS for, in certain instances, the full-year 2026 and fourth quarter of 2026, and future periods, and the underlying drivers of those results, such as the expected demand and continued growth of BioMarin’s Metabolic Conditions portfolio, including PALYNZIQ, GALAFOLD and POMBILITI + OPFOLDA, and VOXZOGO, and the expected impact of the acquisition of Alesta Therapeutics B.V. (Alesta) and the BMN 450 license agreement; the anticipated benefits of the acquisition of Amicus Therapeutics, Inc., including the expected amount and timing of cost synergies as well as expected revenue from the addition of GALAFOLD and POMBILITI + OPFOLDA; the anticipated benefits of the acquisition of Alesta; the anticipated benefits of the BMN 450 license agreement; the anticipated benefits of the global settlement with Ascendis Pharma A/S, including BioMarin’s expected receipt of royalty payments on net sales of Yuviwel in the United States, the European Union, Brazil and South Korea; 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, including with respect to the potential new indication for VOXZOGO in hypochondroplasia; 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 risks and uncertainties include, among others, those factors detailed in BioMarin’s press release issued on November 2, 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 June 30, 2026, 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. 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, 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 Cost of Sales (COS), Non-GAAP Research and Development (R&D) expenses and Non-GAAP Selling, General and Administrative (SG&A) expenses are defined by the company as GAAP COS, 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. Non-GAAP synergies is defined by the company as Amicus’ legacy labor and external spend cost reductions, excluding the impact of stock-based compensation. 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 19 for the reconciliations to the comparable information reported under U.S. GAAP.
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Q3’26 Financial Results Agenda: Key Business Updates Commercial Update Research & Development Update Financial Results 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 Q3’26 Key Business Updates Alexander Hardy Chief Executive Officer
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5 Q3: Execution Across the Business while Investing in the Future ■ 44% Y/Y growth across Metabolic Conditions, led by strong GALAFOLD, POMBILITI + OPFOLDA and VIMIZIM contributions ■ 12% Y/Y growth of VOXZOGO; driven by international expansion and U.S. starts under age 2; large order timing shifted from Q3 to Q4 ■ Record top line and disciplined spend drove significant Non-GAAP Diluted EPS1 growth from underlying operations ■ Seamless commercial integration continues ■ Q3 revenue of $152M for GALAFOLD and $49M for POMBILITI + OPFOLDA ■ Expanding diagnosis and treatment for GALAFOLD and driving switching for POMBILITI + OPFOLDA ■ Cost synergies realization tracking ahead of expectations in 2026; significant cost synergies1,2 expected to be fully realized in 2028 ■ In 2026: Added BMN 450 for Fabry, BMN 440 for hypophosphatasia (HPP), BMN 430 for ReNU syndrome, and BMN 820 for focal segmental glomerulosclerosis (FSGS)3 ■ VOXZOGO for achondroplasia (ACH) full approval PDUFA action date: February 2027 ■ VOXZOGO for hypochondroplasia (HCH) PDUFA action date: May 2027 ■ BMN 351 update expected by YE; BMN 333 and BMN 440 data anticipated in 2027 STRONG Q3’26 PERFORMANCE AMICUS VALUE CREATION BUILDING PIPELINE MOMENTUM +13% GALAFOLD +69% POMBILITI + OPFOLDA $1.025B ▲ 32%Y/Y Total Revenue Growth; First $1B+ Revenue Quarter Q3’26 Patient Growth, Y/Y 1Refer to slide 2 for more detail on Non-GAAP financial measures; 2Represents approximately $280 million of cost reductions on a GAAP basis, and approximately $220 million of cost reductions on a Non-GAAP basis; 3BMN 450 (HM15421/GC1134A), BMN 440 (ALE1), BMN 820 (DMX-200) 4 New Pipeline Assets in 2026; Data and Approvals Expected Across Pipeline
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6 Commercial Update Cristin Hubbard Chief Commercial Officer
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7 Strong GALAFOLD and POMBILITI + OPFOLDA Patient Growth in Q3 GROWTHMetabolic Conditions • +13% Y/Y patient growth • $152M Q3 revenue • Expanded diagnosis and treatment, with deeper penetration across established and newer markets • In international markets, increased KOL engagement and family cascade testing contributed to patient growth • +69% Y/Y patient growth • $49M Q3 revenue • Patient switching supported by a differentiated clinical profile and growing clinical and real-world evidence, including five-year PROPEL data • Underlying growth complemented by new patient starts due to supply constraints for other Pompe therapies
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8 Metabolic Conditions: Diversified Portfolio Drove Record Results $111 $216 $108 $57 $53 $152 $49 GROWTH Q3’26 Dynamics • +44% Y/Y revenue growth, driven largely by the first full quarter of GALAFOLD and POMBILITI + OPFOLDA • Patients on therapy increased across all BioMarin- marketed Metabolic Conditions products, Y/Y • The number of patients on PALYNZIQ increased 9% Y/Y, led by adolescent launch in U.S. • VIMIZIM revenue increased 18% and NAGLAZYME decreased (11%) Y/Y, reflecting international order timing +18% (11%) +6% +10% Metabolic Conditions Q3’26 Revenue +2% $744M, +44% Y/Y1 Metabolic Conditions In millions, except percentages 1Year-over-year revenue comparisons are not available for GALAFOLD and POMBILITI + OPFOLDA because the prior-year periods occurred before BioMarin’s acquisition of Amicus Therapeutics
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9 Skeletal Conditions: Global Growth and Continued Leadership Skeletal Conditions Revenue $218 $245 Q3 2025 Q3 2026 +12% Percentages reflect year-over-year change; 1Dauber, A., Cheung, M.S., Hoover-Fong, J. et al. Diagnosis of hypochondroplasia: international Delphi consensus recommendations. Nat Rev Endocrinol (2026). https://doi.org/10.1038/s41574-026-01296-z In millions, except percentages • ~14,000 total addressable patient population (TAPP) • Focused on sharing strong evidence, increasing diagnosis and patient identification, and laying a foundation for rapid access • HCH diagnosis to benefit from recent international consensus recommendations published in August 2026 in Nature Reviews Endocrinology1 HCH Launch Preparations Q3’26 Dynamics • +12% Y/Y revenue growth; ~17% increase in the number of children treated globally Y/Y • > 75% of revenue generated outside of the U.S. • Large international order previously expected in Q3 now anticipated in Q4 • Children < 2 y/o represented majority of U.S. patient starts
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Research & Development Update Greg Friberg Chief R&D Officer 10
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11 Program Preclinical Phase 1 Phase 2 Phase 3 Key Highlights VOXZOGO Hypochondroplasia • sNDA filing accepted • PDUFA action date: May 30, 2027 BMN 820 (DMX-200) Focal Segmental Glomerulosclerosis (FSGS) • Phase 3 endpoint agreed with FDA • Data expected in 2028 BMN 440 (ALE1) Hypophosphatasia (HPP) • Phase 1/2a ongoing in healthy volunteers and adults with HPP • PK/PD data expected in 2027 BMN 333 Achondroplasia • Phase 2/3 registration-enabling study enrolling; Phase 2 data expected in 2027 BMN 351 Duchenne Muscular Dystrophy • Phase 1/2 enrollment completed • Program update expected by year-end BMN 430 ReNU syndrome • Pre-clinical studies underway • Identifying lead ASO candidate Skeletal Conditions Pipeline ★ New assets added in 2026Metabolic Conditions VOXZOGO Idiopathic Short Stature • Phase 2 data expected in 2027 Pipeline Momentum: Four New Assets in 2026, Multiple Catalysts Ahead BMN 450 (HM15421/GC1134A) Fabry disease POMBILITI + OPFOLDA Late-onset Pompe disease (LOPD) • sNDA filing accepted (for adolescents) • PDUFA action date: Jan. 10, 2027 • Phase 1/2 study ongoing ★ ★ ★ ★
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12 BMN 450: Potential First Subcutaneous ERT for Fabry Disease Long-acting enzyme replacement therapy designed for subcutaneous dosing Target Product Profile BMN 450 has the potential to expand the Fabry market with the addition of a therapy amenable to more patients Pipeline Highlights • Human recombinant α-Gal A designed to extend half-life and improve stability relative to currently available ERTs • Subcutaneous dosing could reduce treatment burden for patients and physicians • Potential to treat all ERT-eligible Fabry patients • Global Phase 1/2 study ongoing; FDA Orphan Drug Designation • Preclinical data show differentiated efficacy in renal function, vascular health and peripheral nervous system outcomes BMN 450
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13 BMN 440 for Hypophosphatasia: Differentiated Mechanism of Action Directly inhibits ENPP1 upstream of PPi, offering a substrate reduction approach for HPP • Oral small molecule targeting ENPP1 to reduce PPi at its source • Potential alternative to ERT for HPP patients (both pediatric + adult-onset) • Potential to address systemic symptoms beyond bone, including muscle weakness, pain and fatigue ENPP1 hydrolyzes ATP to PPi and AMP BMN 440 Deficient alkaline phosphatase activity causes PPi accumulation, impairing bone mineralization and contributing to musculoskeletal pain and weakness Target Product Profile BMN 440 has the potential to be the first oral therapy for HPP, including adults who are largely untreated today Pipeline Highlights • Phase 1/2a ongoing in healthy volunteers and adults with HPP • Data readout expected in 2027
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14 POMBILITI + OPFOLDA Demonstrates Durable Outcomes Through Five Years in LOPD Key Highlights Biomarker improvement: Sustained serum CK reductions in both ERT-experienced and ERT- naïve patients. Long-term pulmonary stability: % predicted sitting FVC remained relatively stable in ERT- experienced and ERT-naïve patients. Consistent long-term safety through Year 5, consistent with published 2-year data. Long-term durability across motor function, pulmonary function, biomarker response, and safety Roberts M, et al. Long-term (5-year) efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease treated from PROPEL baseline. Poster presented at: World Muscle Society (WMS) Annual Congress; September 29–October 3, 2026; Hiroshima, Japan. Cipa+mig Switch
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15 Q3’26 Financial Results Brian Mueller Chief Financial Officer
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16 Q3 Financial Highlights Q3 2026 Results Non-GAAP Diluted EPS • $0.23 EPS, +92% Y/Y, primarily driven by revenue growth, partially offset by higher acquired IPR&D charges and interest expense 1Refer to slide 2 for more detail on Non-GAAP financial measures; 2N.M. = not meaningful Total Revenues • $1,025M, +32% Y/Y, primarily driven by addition of GALAFOLD and POMBILITI + OPFOLDA • Royalty and Other Revenues now include royalties from Ascendis Operating Expenses • Acquired IPR&D expenses are primarily related to Alesta acquisition • Non-GAAP SG&A increased Y/Y, driven by addition of sales and marketing spend from Amicus to support the growth of GALAFOLD and POMBILITI + OPFOLDA In millions, except percentages and per share amounts Q3’26 Y/Y Change Total Revenues $1,025 +32% GAAP R&D $181 (4%) Acquired IPR&D $288 +30% GAAP SG&A $334 +24% GAAP Operating Margin (8.0%) (2.0) ppts Non-GAAP R&D1 $167 (4%) Non-GAAP SG&A1 $249 +12% Non-GAAP Operating Margin1 13.4% +10.6 ppts GAAP Diluted EPS ($0.63) +294% Non-GAAP Diluted EPS1 $0.23 +92% Interest Income $5 (74%) Interest Expense ($56) N.M.2
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17 Updated Full-year 2026 Guidance (In millions, except per share amounts) Prior Guidance As of August 6, 2026 Updated Guidance As of November 2, 2026 Midpoint Growth (Y/Y) Total Revenues $3,875 to $3,925 $3,800 to $3,900 20% Metabolic Conditions $2,725 to $2,775 $2,675 to $2,750 29% VOXZOGO $1,000 to $1,050 $975 to $1,025 8% Other Revenues1 $100 to $125 $125 to $150 Non-GAAP Diluted EPS2,3 $4.90 to $5.10 $2.80 to $3.10 (6%) 1Other Revenues includes KUVAN, ROCTAVIAN, and royalties. 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 • Revenue: Total Revenues, Metabolic Conditions, and VOXZOGO guidance updated to reflect uncertainty in the timing of multiple orders from a single international customer. • Non-GAAP Diluted EPS: BioMarin expects the Alesta acquisition and BMN 450 license agreement to result in total pre-tax charges of approximately $320 million and $83 million, respectively, for the full year 2026, consisting of the acquired IPR&D charges and ongoing operating expenses, representing an unfavorable impact of approximately $1.55 and $0.32, respectively, to both GAAP Diluted EPS and Non-GAAP Diluted EPS for the full year 2026.
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18 Q&A
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Appendix Reconciliation of GAAP Reported to Selected Non-GAAP Adjusted Information
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20 Reconciliation of GAAP Reported Net Income (Loss) to Non-GAAP Income(1) (In millions) Please refer to slide 24 for footnotes to the table
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21 Reconciliation of GAAP and Non-GAAP COS, R&D, and SG&A Expenses(1) (In millions) Please refer to slide 24 for footnotes to the table
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22 Reconciliation of GAAP Income (Loss) from Operations to Non-GAAP Income from Operations(1) (In millions, except percentages) Please refer to slide 24 for footnotes to the table
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23 Reconciliation of GAAP Diluted EPS to Non-GAAP Diluted EPS(1) (In U.S. dollars) Please refer to slide 24 for footnotes to the table
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24 Reconciliation of GAAP Weighted-Average Diluted Shares Outstanding to Non-GAAP Weighted-Average Diluted Shares Outstanding(1) (In millions) (1) Certain amounts may not sum or recalculate due to rounding. (2) Stock-based compensation expense recorded in SG&A for the nine months ended September 30, 2026, includes approximately $13 milli on related to the post-combination service period for unvested Amicus stock options. (3) Represents amortization of the inventory step-up for the inventory acquired in the Amicus transaction. (4) The 2026 amounts represent severance, transaction and integration costs resulting from the Amicus acquisition, as well as restructuring costs incurred in connection with corporate initiatives. The 2025 amounts represent severance costs incurred in connection with the acquisition of Inozyme Pharma, Inc. (5) Represents impairment loss on non-marketable equity securities recorded in Other income, net, in the first quarter of 2025. (6) Common stock issuable under the company’s equity plans and convertible debt were excluded from the computation of GAAP and Non-GAAP Weighted-Average Diluted Shares Outstanding in the periods they were anti-dilutive.