On October 27, 2025 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) reported financial results for the third quarter ended September 30, 2025.
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"We are pleased with the contributions from our Enzyme Therapies and Skeletal Conditions business units to date this year driven by more than 20% revenue growth from PALYNZIQ and VOXZOGO," said Alexander Hardy, President and Chief Executive Officer of BioMarin. Our strategic investments in these focused business units are generating strong results, and we anticipate sustained financial performance from each of them. Both Enzyme Therapies and Skeletal Conditions remain central to our growth strategy, in addition to new business development opportunities and our advancing internal pipeline. As we focus on the business units aligned with our strategic priorities, today we are announcing the decision to pursue options to divest ROCTAVIAN and remove it from our portfolio. We continue to believe ROCTAVIAN has an important role to play in the treatment of hemophilia A and are therefore evaluating out-licensing options for this innovative gene therapy. This decision is consistent with BioMarin’s portfolio strategy and offers the most promising opportunity for ensuring continued patient access to ROCTAVIAN.
Mr. Hardy concluded, "Looking ahead, we will rely on our disciplined strategic focus and proven capabilities to develop and commercialize innovative therapies that generate sustainable value for patients, employees, and shareholders."
Third Quarter 2025 Financial Highlights
•Total Revenues for the third quarter of 2025 were $776 million, an increase of 4% compared to the same period in 2024, driven by strong revenue growth in VOXZOGO and PALYNZIQ attributable to new patients initiating therapy across all regions. These increases were partially offset by lower sales volume for ALDURAZYME due to timing of order fulfillment to Sanofi and NAGLAZYME due to timing of large government orders in Latin America.
•GAAP Net Loss increased to $31 million for the third quarter of 2025 compared to GAAP Net Income of $106 million for the same period in 2024. The increase in GAAP Net Loss was primarily due to the In-Process Research & Development (IPR&D) charge of $221 million recorded in connection with the acquisition of Inozyme Pharma, Inc. (Inozyme). This increase in GAAP Net Loss was partially offset by improved gross profit as a result of revenue growth mentioned above, lower Cost of Sales from favorable product mix during the quarter, and lower provision for income taxes.
•Non-GAAP Income for the third quarter of 2025 decreased to $22 million compared to $178 million for the same period in 2024. The decrease in Non-GAAP Income was primarily due to the factors noted above.
Third Quarter 2025 Business Highlights
Innovation
•Skeletal Conditions: At the American Society for Bone and Mineral Research (ASBMR) Annual Meeting, investigators shared new data demonstrating improved spinal morphology – one of the factors that contributes to spinal stenosis, a leading cause of morbidity in achondroplasia – following treatment with VOXZOGO in children ages 5 and under (see PR here). VOXZOGO is the only approved therapy with data showing a positive impact on spinal morphology, and these findings add to the extensive body of evidence supporting VOXZOGO’s health benefits beyond improving growth.
•As a follow-up to the encouraging PK data announced last quarter for BMN 333, BioMarin’s long-acting C-type natriuretic peptide, the company plans to initiate dosing of children with achondroplasia in its registration-enabling Phase 2/3 study in the first half of 2026.
•VOXZOGO pivotal data for the treatment of hypochondroplasia is expected in the first half of 2026, followed by potential launch in 2027, should data be supportive. Hypochondroplasia can be associated with significant co-morbidities, including respiratory, orthopedic, mental health, and ear nose and throat complications, representing high unmet medical need for this skeletal condition with no approved therapies.
•Four new VOXZOGO indications are under development as part of BioMarin’s CANOPY clinical program, with a focus on the most severely impacted sub-set of children. These conditions include idiopathic short stature, Noonan syndrome, Turner syndrome, and SHOX deficiency, and are currently enrolling patients, with potential registration-enabling studies in 2027.
•Enzyme Therapies: Based on new data from the PALYNZIQ Phase 3 PEGASUS study in 12- to 17-year-olds demonstrating statistically significant blood phenylalanine (Phe) lowering compared to diet alone, the company is pursuing approvals in this age group in the United States and Europe, with potential approval in 2026.
•With BMN 401, a potential first-in-disease treatment for ENPP1 deficiency, initial pivotal data readout for the ENERGY 3 study in children ages 1–12 years is anticipated in the first half of 2026, with potential launch in 2027.
•Other Clinical Updates: For BMN 351, BioMarin’s next generation oligonucleotide for Duchenne muscular dystrophy, the company expects to share a clinical update for the 6 mg/kg and 9 mg/kg cohorts by year-end.
Growth
•As of the end of the quarter, children with achondroplasia in 55 countries around the world were being treated with VOXZOGO, tracking to the company’s plan to open access in more than 60 countries by 2027. Year-to-date VOXZOGO revenue increased 24% Y/Y, with Q4’25 VOXZOGO revenue expected to reach its highest level of the year. BioMarin reaffirmed full-year 2025 VOXZOGO revenue outlook of between $900 million and $935 million.
•Representing approximately 75% of total VOXZOGO revenue, markets outside of the U.S. (OUS) benefited from BioMarin’s established global footprint to drive VOXZOGO uptake across key large markets during the quarter.
•Initiatives implemented to expand treatment with VOXZOGO in the U.S. resulted in new patient starts across all ages in the third quarter, with the majority from children under 2 years of age. Due to the geographical dispersion and management across a range of specialties for older children in the U.S, the company has implemented initiatives to address slowing uptake in that demographic.
•PALYNZIQ marked its third consecutive quarter of 20%+ Y/Y growth. PALYNZIQ strength continued to be driven by greater numbers of patients titrating to daily maintenance dose and strong adherence. Total Enzyme Therapies revenue grew 8% Y/Y, year-to-date, reflecting high penetration rates and strong adherence to these treatments.
•Today, the company announced its plan to pursue options to divest ROCTAVIAN, including exploring out-licensing opportunities. BioMarin plans to continue to make ROCTAVIAN commercially available in the U.S., Italy and Germany until next steps are finalized. The company will continue to provide support and monitoring for people treated with ROCTAVIAN.
Value Commitment
•Acquired IPR&D charges in Q3 from BioMarin’s acquisition of Inozyme resulted in Y/Y increases in GAAP and Non-GAAP R&D expenses. Q3 GAAP and Non-GAAP SG&A expenses increased Y/Y due to investment in business unit expansion initiatives. Year-to-date GAAP Diluted EPS and Non-GAAP Diluted EPS increased Y/Y, driven by underlying strong revenue performance and operational efficiencies.
•The company generated operating cash flows totaling $369 million in third quarter 2025 and generated $728 million in year-to-date operating cash flows. Total cash and investments at the end of the third quarter were approximately $2.0 billion, and increasing operating cash flow is expected to continue, supporting BioMarin’s priority of investment in innovation and future growth.
•Today, BioMarin increased total revenue guidance, at the midpoint, reflecting strong demand for its therapies through 2025. Revised Non-GAAP Operating Margin and Non-GAAP Diluted EPS guidance include the impact of Q3 acquired IPR&D expenses, partially offset by underlying strong topline performance and operational execution throughout the year.
Refer to the 2025 Full-Year Financial Guidance on page 4 of this press release.
Financial Highlights (in millions of U.S. dollars, except per share data, unaudited)
Three Months Ended
September 30, Nine Months Ended
September 30,
2025 2024 % Change 2025 2024 % Change
Total Revenues $776 $746 4% $2,347 $2,107 11%
Net Product Revenues by Product:
VOXZOGO $218 $190 15% $654 $527 24%
Enzyme Therapies:
VIMIZIM $183 $178 3% $587 $549 7%
NAGLAZYME 122 132 (8)% 365 370 (1)%
PALYNZIQ 109 91 20% 308 255 21%
ALDURAZYME 54 71 (24)% 159 145 10%
BRINEURA 48 37 30% 137 121 13%
Total Enzyme Therapies Revenue $516 $509 1% $1,556 $1,440 8%
KUVAN $24 $28 (14)% $76 $93 (18)%
ROCTAVIAN
$3 $7 (57)% $23 $16 44%
GAAP Net Income (Loss)(1)
$(31) $106 (129)% $395 $302 31%
Non-GAAP Income (1)(2)
$22 $178 (88)% $525 $506 4%
GAAP Operating Margin % (1)(3)
(6.0)% 15.3% 19.4% 15.3%
Non-GAAP Operating Margin % (1)(2)
2.8% 27.7% 26.3% 27.7%
GAAP Diluted Earnings (Loss) per Share (EPS)(1)
$(0.16) $0.55 (129)% $2.04 $1.56 31%
Non-GAAP Diluted EPS (1)(2)
$0.12 $0.91 (87)% $2.69 $2.60 3%
September 30,
2025 December 31,
2024
Total cash, cash equivalents & investments $ 1,991 $ 1,659
(Press release, BioMarin, OCT 27, 2025, View Source [SID1234657031])