Slides
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Second Quarter 2026 Financial Results August 6 , 2026 BIOMARIN 1
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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 Total Revenues, Non-GAAP Diluted EPS, Non-GAAP Operating Margin, gross leverage, operating cash flow and revenue compound annual growth rate (CAGR) for, in certain instances, the full-year 2026, fourth quarter and second half 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, and VOXZOGO, and the expected impact of the acquisition of Amicus Therapeutics, Inc. (Amicus); the anticipated benefits of the acquisition of Amicus, including the expected amount and timing of cost synergies as well as expected revenue from the addition of GALAFOLD and POMBILITI + OPFOLDA, including BioMarin’s plans and expectations to accelerate growth through mid-2030s; 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 August 6, 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 March 31, 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. Projected Gross Leverage is defined by the company as undiscounted debt (total debt excluding unamortized discount and deferred offering costs) as of the balance sheet date divided by 4 quarter projected Non-GAAP Adjusted EBITDA. Non-GAAP Adjusted EBITDA is defined by the company as GAAP Income (or Loss) from Operations excluding the impact of depreciation, amortization and stock-based compensation expense. 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 22 for the reconciliations to the comparable information reported under U.S. GAAP.
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Q2’26 Financial Results Agenda: Q2’26 Results and Amicus Integration Update 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 Q2’26 Key Business and Amicus Integration Updates Alexander Hardy Chief Executive Officer
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5 Q2: Strengthened Rare Disease Leadership $990M ▲ 20%Y/Y Total Revenue Growth ■ 25% Y/Y growth across Metabolic Conditions (formerly Enzyme Therapies), including GALAFOLD and POMBILITI + OPFOLDA ■ 14% Y/Y growth of VOXZOGO; > 20% Y/Y increase in number of children treated; strong U.S. and global demand led to increased full-year guidance of at least $1 billion ■ FY’26 Non-GAAP Diluted EPS guidance increased; Total Revenues midpoint raised ■ Q4 expected to be highest revenue quarter in 2026 ■ Rapid, value-creating integration of Amicus, following April 27 close ■ Leverages BioMarin’s global scale and capabilities platform to drive growth ■ Significant cost synergies1,2 expected to be fully realized in 2028 ■ Increasing operating cash flow expected; substantial EPS accretion anticipated beginning in 2027 ■ Accelerated de-leveraging, now targeting < 2.5x gross leverage1 by mid-year 2027 Phase 3 Win Hypochondroplasia (HCH) Potential to Expand VOXZOGO Market ■ sNDA submitted for the approval of VOXZOGO for HCH based on strong pivotal data ■ 5-year leadership in achondroplasia (ACH) facilitates potential launch of first targeted therapy for HCH ■ BMN 820 (formerly DMX-200) Phase 3 ongoing; first-in-class oral CCR2 for FSGS; pivotal data expected 2028 ■ U.S. and EU approval in adolescents broadens PALYNZIQ opportunity ■ Plans to fortify earlier-stage pipeline in next 12 to 18 months Accelerated Growth Forward-looking figures are company estimates based on current portfolio; Estimated peak revenue for GALAFOLD expected by the mi d-2030s and for POMBILITI + OPFOLDA by the mid-to-late-2030s; 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, expected to be fully realized in 2028, representing an approximately 50% reduction from Amicus-reported 2025 GAAP and Non-GAAP operating expenses, respectively. STRONG Q2’26 EXECUTION AMICUS VALUE CREATION PIPELINE MOMENTUM ~$1.4B GALAFOLD Peak Revenue ~$1.2B POMBILITI + OPFOLDA Peak Revenue
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6 Accelerating BioMarin’s Growth Trajectory through Mid-2030s • Significant Cost Synergies Expected to be Fully Realized in 2028 • GALAFOLD and POMBILITI + OPFOLDA Peak Revenue • Expanding Combined Portfolio Non-GAAP Operating Margin • Generating Significant Operating Cash Flow
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7 Accelerating the GALAFOLD Peak Revenue Outlook 2025A Driving diagnosis & treatment Increased penetration & global expansion Mid 2030s Peak Revenue Estimate ~10% CAGR ('27-'32) Growth and Acceleration Levers Driving Diagnosis and Treatment • Globally, scale AI-enabled patient finding, genetic testing, newborn screening, and family cascade screening • Goal to more than double number of U.S. patients treated with GALAFOLD (~40% of 2025 revenue from U.S.) Driving Penetration and Global Expansion • Goal to treat additional patients in established markets • Expand or enter more than 10 priority markets where BioMarin has established commercial capabilities Driving Diagnosis and Treatment Driving Penetration and Global Expansion 2025A Mid- 2030s $1.4B $522M
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8 Accelerating the POMBILITI + OPFOLDA Peak Revenue Outlook ≥ 20% CAGR ('27-'32) Growth and Acceleration Levers Driving Diagnosis and Treatment • Leverage BioMarin’s diagnostic capabilities to identify naïve- to-treatment patients within global footprint Driving Switch and Global Expansion • HCP and patient education; real-world-evidence generation to demonstrate the benefit of switching • Leverage AI patient finding model that identifies patients waning on current therapy • Increase number of U.S. treated patients by > 7x • Goal to establish more than 20 new markets over time Driving Switch and Global Expansion Driving Diagnosis and Treatment Mid-to-late 2030s 2025A Driving switch & global expansion Driving diagnosis & treatment Mid-to-late 2030s $113M Peak Revenue Estimate $1.2B 2025A
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9 ~$220M in Amicus Cost Synergies1,2 Expected to Result in ~50% Reduction from Amicus-reported 2025 Non-GAAP Operating Expenses1 Cost Synergies Split ~$220M Annual Run-Rate Cost Reduction, in 2028 G&A: > 70% R&D / Other: < 30% Key Synergies Drivers • Significant reductions in R&D and G&A • S&M retained; maintaining HCP and patient- facing capabilities to drive commercial expansion • Expected to realize full cost reduction annual run rate of ~$220M in 2028 • ~$220M represents ~50% reduction from Amicus- reported 2025 Non-GAAP Operating Expenses of $432M 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, expected to be fully realized in 2028, representing an approximately 50% reduction from Amicus-reported 2025 GAAP and Non-GAAP operating expenses, respectively.
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10 Creating Significant Value Near-term and Beyond 2027 2028 Beyond 2030 Cost Synergies1,2 Ramping Gross Leverage1 < 2.5x (mid-year 2027) Cost Synergies Fully Realized ~$220M Annual run-rate cost reduction ~50% Reduction from Amicus-reported 2025 Non- GAAP Operating Expenses1 of $432M Peak Revenue Outlook $1.4B by mid-2030s (~10% CAGR ’27-’32) $1.2B by mid-to-late-2030s (≥ 20% CAGR ’27-’32) Substantial Non-GAAP Diluted EPS1 Accretion Expected Beginning in 2027 Significant Increase in Operating Cash Flow Anticipated GALAFOLD and POMBILITI + OPFOLDA Combined Non-GAAP Operating Margin1 > 60% All figures are company estimates based on current portfolio. All figures represent additive contribution from Amicus acquisition, except Gross Leverage, which is estimated for combined company; 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, expected to be fully realized in 2028, representing an approximately 50% reduction from Amicus-reported 2025 GAAP and Non-GAAP operating expenses, respectively. 2 GALAFOLD POMBILITI + OPFOLDA
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11 Commercial Update Cristin Hubbard Chief Commercial Officer
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12 Acceleration of Galafold and Pombiliti + Opfolda Underway GROWTHMetabolic Conditions $111 $129 Q2 2024A Q2 2025A Q2 2026A $16 $26 Q2 2024A Q2 2025A Q2 2026A +62% + > 65% $ in millions Revenue $ in millions Revenue Charts are not to scale and illustrative only; Percentages reflect year-over-year change; 1Q2’26 revenue growth calculated from unaudited pro-forma figures and includes revenue from April 1, 2026 to April 26, 2026, prior to the acquisition of Amicus 1 1 +16% FY 2024A: $458M FY 2025A: $522M FY 2024A: $70M FY 2025A: $113M + ~10%
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13 Metabolic Conditions (Formerly Enzyme Therapies): More Diversified Portfolio Drove Strong Q2’26 $135 $194 $135 $44 $51 $106 $30 GROWTH Q2’26 Dynamics • 25% Y/Y revenue growth, inclusive of GALAFOLD and POMBILITI + OPFOLDA • Patients on therapy grew across all BioMarin-marketed Metabolic Conditions, both Y/Y and sequentially • PALYNZIQ revenue grew 27% Y/Y, driven by continued patient demand, including from ages 12+, and favorable order timing in the U.S. • European Commission approved PALYNZIQ for adolescents 12 years and older with PKU • Order timing in Q1 impacted VIMIZIM; full-year guidance remains the best indicator of underlying performance (10%) +5% (21%) +4% Metabolic Conditions Q2’26 Revenue 27% $695M, +25% Y/Y Metabolic Conditions $ in millions Reported revenue for April 27 – June 30
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14 Skeletal Conditions: Strong Demand in Q2’26 for VOXZOGO for ACH Skeletal Conditions Revenue $221 $253 Q2 2025 Q2 2026 +14% Percentages reflect year-over-year change $ in millions • sNDA submitted to FDA for the treatment of hypochondroplasia • In July, FDA accepted sNDA for full approval in achondroplasia Pipeline Highlights Q2’26 Dynamics • Double-digit U.S. and OUS revenue growth and strong 2H outlook led to increased FY’26 VOXZOGO guidance • Approximately three-quarters of VOXZOGO revenue generated OUS • Children treated increased > 20% Y/Y globally • > 50% of new U.S. patient starts were in < 2 y/o age group; expect continued leadership in incident market • Revenue expected to increase in 2H vs. 1H
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Research & Development Update Greg Friberg Chief R&D Officer 15
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16 2026 R&D Highlights: Recent Updates and Anticipated Milestones Includes both completed / ongoing and anticipated R&D highlights (non-exhaustive) Program Status and Upcoming Milestones Recent Updates Completed in April – July 2026 ✓ VOXZOGO Phase 3 topline data for hypochondroplasia; sNDA submitted ✓ VOXZOGO sNDA for full approval in achondroplasia accepted by FDA (PDUFA date: Feb. 28, 2027) ✓ BMN 333 Phase 1 PK & PD data presented at ENDO Candidate Condition Key Highlights Hypochondroplasia • sNDA submitted; subject to FDA review and acceptance • Phase 3 full data at ESPE in September (late-breaking oral presentation) • Potential first-in-class launch in 2027 Idiopathic Short Stature • Phase 2 study progressing Noonan Syndrome • Phase 2 study progressing BMN 333 Achondroplasia • Phase 2/3 registration-enabling study enrolling BMN 351 Duchenne muscular dystrophy • Phase 1/2 enrollment completed • Program update expected by year-end BMN 820 (formerly DMX-200) Focal Segmental Glomerulosclerosis • Phase 3 study advancing ✓ PALYNZIQ EU approval received for adolescents 12 years and older with phenylketonuria ✓ VOXZOGO 3-year extension data (investigator sponsored study) at ENDO demonstrate sustained growth in children with hypochondroplasia
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17 Potential Upside with BMN 820 for Focal Segmental Glomerulosclerosis (FSGS); Phase 3 Endpoint Agreed with FDA FSGS Background and BMN 820 Opportunity FSGS Background • FSGS is a progressive kidney disease in which scarring of the kidney's filtering units causes protein in the urine and declining kidney function over time • Only one therapy is approved today, with a narrow label that excludes FSGS patients with nephrotic syndrome • Durable stabilization of kidney function and complete remission, consistent with international KDIGO2 treatment guidelines, remain significant unmet need BMN 820 Opportunity • First-in-class oral CCR2 inhibitor with potential to treat a broad FSGS population, including primary, genetic, and undetermined FSGS, regardless of nephrotic syndrome status • Favorable safety and tolerability profile to date • Studied in combination with background ARB therapy, a standard of care in FSGS • FDA has agreed that proteinuria is an appropriate endpoint for full approval for BMN 820 in ACTION3 (Phase 3 trial) • Phase 3 data expected in 2028 BMN 820 Total Addressable Patient Population1 in the United States: ~30,000 1Total addressable patient population (TAPP) defined as diagnosed FSGS patients ages 12 to 80 of primary, genetic, or undeterm ined cause, regardless of therapy status; 2Kidney Disease: Improving Global Outcomes
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18 Q2’26 Financial Results Brian Mueller Chief Financial Officer
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19 Q2 Financial Highlights Q2 2026 Results In millions, except percentages and per share amounts Q2’26 Y/Y Change Total Revenues $990 +20% GAAP R&D $207 +28% GAAP SG&A $396 +70% GAAP Operating Margin 11.2% (22.3) ppts Non-GAAP R&D1 $191 +30% Non-GAAP SG&A1 $253 +25% Non-GAAP Operating Margin1 36.4% (3.5) ppts GAAP Diluted EPS $0.23 (81%) Non-GAAP Diluted EPS1 $1.20 (17%) Interest Income $10 (44%) Interest Expense $63 NM Non-GAAP Diluted EPS • Higher operating expenses and acquisition financing costs drove lower Y/Y EPS 1Refer to slide 2 for more detail on Non-GAAP financial measures Non-GAAP R&D and SG&A • Y/Y increases reflect Amicus operating expenses, continued pipeline investment and commercial execution • GAAP SG&A includes ~$84M of transaction- and integration-related charges associated with the Amicus acquisition, which are excluded from Non-GAAP results Interest Expense and Interest Income • Based on current rates, interest expense associated with Amicus financing is estimated at approximately $200 million on an annualized basis • Due to cash and investments used to fund the acquisition, interest income expected to decrease year-over-year, in near-term
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20 Updated Full-year 2026 Guidance (In millions, except per share amounts) Prior Guidance As of May 4, 2026 Updated Guidance As of August 6, 2026 Midpoint Growth (Y/Y) Total Revenues $3,825 to $3,925 $3,875 to $3,925 21% Metabolic Conditions $2,725 to $2,775 Unchanged 31% VOXZOGO $975 to $1,025 $1,000 to $1,050 11% Other Revenues1 $100 to $125 Unchanged Non-GAAP Diluted EPS2,3,4 $4.85 to $5.05 $4.90 to $5.10 59%5 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; 4Non-GAAP Diluted EPS guidance assumes a combined company tax rate of 22%; 52025 Non-GAAP Diluted EPS included acquired in-process research & development charges related to the acquisition of Inozyme and an inventory write-off related to the company's strategic decision to voluntarily withdraw ROCTAVIAN from the market Total Revenues, VOXZOGO, and Non-GAAP Diluted EPS guidance raised, reflecting strong first-half 2026 performance and second-half 2026 revenue expectations for both Metabolic Conditions and VOXZOGO
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21 Q&A
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Appendix Reconciliation of GAAP Reported to Selected Non-GAAP Adjusted Information
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23 Reconciliation of GAAP Reported Net Income to Non-GAAP Income(1) (In millions of U.S. dollars) Please refer to slide 27 for footnotes to the table
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24 Reconciliation of GAAP and Non-GAAP COS, R&D, and SG&A Expenses(1) (In millions of U.S. dollars) Please refer to slide 27 for footnotes to the table
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25 Reconciliation of GAAP Income from Operations to Non-GAAP Income from Operations(1) (In millions of U.S. dollars) Please refer to slide 27 for footnotes to the table
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26 Reconciliation of GAAP Diluted EPS to Non-GAAP Diluted EPS(1) (In U.S. dollars) Please refer to slide 27 for footnotes to the table
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27 Reconciliation of GAAP Weighted-Average Diluted Shares Outstanding to Non-GAAP Weighted-Average Diluted Shares Outstanding(1) (In U.S. dollars) (1) Certain amounts may not sum or recalculate due to rounding. (2) Stock-based compensation expense recorded in SG&A for the three and six months ended June 30, 2026, includes approximately $13 m illion related to the post-combination service period for unvested Amicus stock options. (3) These amounts represent costs resulting from the Amicus acquisition that closed on April 27, 2026. Acquisition-related costs were included in SG&A and consisted of severance, transaction and integration costs. Amortization of acquired inventory -up was included in COS. (4) These amounts were included in SG&A and represent charges for severance in connection with the company’s plan to simplify its organizational design and strategic initiatives in the first and second quarters of 2026. (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 convertible debt were excluded from the computation of GAAP Weighted-Average Diluted Shares Outstanding for the three and six months ended June 30, 2026 as they were anti-dilutive.
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28 Amicus Therapeutics, Inc.(1) Reconciliation of Non-GAAP Financial Measures (In thousands of U.S. dollars) (1) The above historical reconciliation is reproduced from Amicus’ earnings release furnished as Exhibit 99.1 to its Current Report on Form 8-K dated February 20, 2026 and reflects Amicus’ historical definitions of the applicable non-GAAP measures.