On November 2, 2026 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) reported financial results for the third quarter ended September 30, 2026.

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"BioMarin delivered a strong quarter, with revenue growth of 32%, driven by a more diversified portfolio following the acquisition of Amicus Therapeutics earlier this year and increased demand for our medicines from patients around the world. For the first time, quarterly revenue exceeded $1 billion, a milestone for BioMarin," said Alexander Hardy, President and Chief Executive Officer of BioMarin. "We are building on this achievement by continuing to expand our pipeline through focused business development. We are excited to add our newest asset, an investigational treatment for Fabry disease, which has the potential to become the first subcutaneous enzyme therapy for this condition. With this fourth new asset added to our pipeline in 2026, we are following through on our commitment to build sustainable growth for the future." Mr. Hardy added, "As we approach 2027, we are well positioned for our next chapter of growth, including the potential launch of VOXZOGO in hypochondroplasia and the continued execution of our financial and innovation strategy."

2026 Business and Pipeline Highlights
Innovation
•BioMarin is accelerating innovation by expanding its pipeline through business development. Four transactions in 2026 have added development programs from preclinical through Phase 3:

◦BMN 450 (HM15421/GC1134A) is an investigational long-acting enzyme replacement therapy (ERT) that has the potential to be the first subcutaneous ERT for Fabry disease. Now in a global Phase 1/2 clinical study, it could extend BioMarin’s reach beyond patients with genetic variants amenable to GALAFOLD.

◦BMN 440 (ALE1) for hypophosphatasia (HPP) is a potential first-in-class oral therapy for HPP. The Phase 1/2a study in healthy volunteers and adults with HPP is ongoing, with data expected in 2027.

◦BMN 430 for ReNU syndrome has the potential to be a first-in-disease antisense oligonucleotide (ASO) medicine for ReNU syndrome, a serious, rare neurodevelopmental condition with no approved targeted therapies. Preclinical studies are underway to identify a lead ASO candidate.

◦BMN 820 (DMX-200) for focal segmental glomerulosclerosis (FSGS) is a first-in-class oral CCR2 inhibitor for which BioMarin holds exclusive U.S. commercialization rights. The Phase 3 ACTION 3 trial is fully enrolled, with pivotal data expected in 2028.

•Today, BioMarin announced that the U.S. Food and Drug Administration (FDA) has set a PDUFA target action date of May 30, 2027 for its supplemental NDA to expand the use of VOXZOGO for the treatment of HCH, the first potential targeted therapy for the condition. BioMarin also announced today that the European Medicines Agency (EMA) validated its submission for the approval of VOXZOGO to treat HCH.

•BioMarin’s application for full approval of VOXZOGO in children with achondroplasia is under review by the FDA, with a PDUFA target action date of February 28, 2027. The application includes long-term safety and efficacy data, such as final adult height and clinical outcomes beyond linear growth.

•The Phase 2/3 study for BMN 333, BioMarin’s long-acting C-type natriuretic peptide (CNP) for achondroplasia, continues to enroll on track. Dose-response data from this study is expected in 2027.

•In October, five-year results from the PROPEL open-label extension study with POMBILITI + OPFOLDA were presented in a late-breaking presentation at the World Muscle Society Annual Congress. In adults with late-onset Pompe disease, POMBILITI + OPFOLDA demonstrated durable motor function outcomes and relative stabilization of pulmonary function. This included people who had received enzyme replacement therapy for a mean of 7.6 years before enrolling. No new safety signals were identified.
•For BMN 351, BioMarin’s Phase 1/2 candidate for Duchenne muscular dystrophy, the company expects to provide a program update by year-end.
Growth
•In the first full quarter as part of BioMarin, GALAFOLD and POMBILITI + OPFOLDA delivered strong quarterly revenue, contributing $152 million and $49 million, respectively. The number of patients treated with GALAFOLD and POMBILITI + OPFOLDA increased by approximately 13% and 69% Y/Y, respectively. POMBILITI + OPFOLDA uptake benefited from supply constraints for other Pompe therapies.
•Metabolic Conditions revenue grew 44% Y/Y in the third quarter of 2026 and 26% Y/Y year-to-date. Growth was driven by the additions of GALAFOLD and POMBILITI + OPFOLDA and by higher VIMIZIM revenue. The number of patients on therapy increased across all BioMarin-marketed Metabolic Conditions therapies, Y/Y.
•VOXZOGO revenue grew 12% Y/Y in the third quarter of 2026, and the number of children treated with VOXZOGO globally increased 17% Y/Y. As of the end of the quarter, children with achondroplasia in 58 countries around the world were being treated with VOXZOGO. More than 75% of VOXZOGO revenue was generated outside of the U.S., and a large international order previously expected in the third quarter is now anticipated in the fourth quarter. In the U.S., the majority of new patient starts were in children under the age of two.

Value Commitment
•BioMarin continued to integrate Amicus in the third quarter, realizing accelerated cost synergies to enable investments in initiatives to drive future growth of GALAFOLD and POMBILITI + OPFOLDA. BioMarin continues to expect approximately $280 million of GAAP cost synergies (approximately $220 million on a Non-GAAP basis) to be fully realized in 2028.
•On August 30, 2026, the company announced the global settlement of all outstanding claims with Ascendis Pharma A/S related to its product Yuviwel. As part of the agreement, Ascendis will pay BioMarin a royalty equal to 20% of net sales of Yuviwel in the U.S., retroactive to the first commercial sale, and 18% of net sales in the European Union, Brazil and South Korea until May 2030.

Third Quarter 2026 Financial Highlights
•Total Revenues for the third quarter of 2026 were $1,025 million, an increase of 32% compared to the same period in 2025, primarily driven by revenues from GALAFOLD and POMBILITI + OPFOLDA, which were acquired on April 27, 2026, as well as 18% revenue growth in VIMIZIM due to timing of large international orders. In the quarter, VOXZOGO revenue also increased by 12% due to new patients initiating therapy. These increases were partially offset by a decrease in NAGLAZYME revenue due to timing of large international orders.

•GAAP Net Loss increased to $123 million compared to $31 million for the same period in 2025. The increase was primarily driven by the acquisition of Amicus Therapeutics (Amicus), including intangible asset amortization, interest expense from debt issued to finance a portion of the transaction, amortization of inventory fair value step-up, and integration and restructuring costs. The increase was also attributed to acquired in-process research and development (IPR&D) charges recorded in the quarter in connection with the acquisition of Alesta Therapeutics (Alesta). These increases were partially offset by revenue growth as described above.

•Non-GAAP Income for the third quarter of 2026 increased to $45 million compared to $22 million for the same period in 2025. The increase was primarily driven by revenue growth, partially offset by higher acquired IPR&D charges and higher interest expense, as described above. The increases were also partially offset by higher sales and marketing spend related to GALAFOLD and POMBILITI + OPFOLDA.

Financial Highlights (in millions of U.S. dollars, except percentages and per share data, unaudited)
Three Months Ended
September 30, Nine Months Ended
September 30,
2026 2025 % Change 2026 2025 % Change
Total Revenues $1,025 $776 32% $2,781 $2,347 18%
Net Product Revenues by Product:
VOXZOGO $245 $218 12% $717 $654 10%
Metabolic Conditions:
VIMIZIM $216 $183 18% $620 $587 6%
NAGLAZYME 108 122 (11)% 373 365 2%
PALYNZIQ
111 109 2% 336 308 9%
GALAFOLD 152 — NM 258 — NM
BRINEURA
53 48 10% 151 137 10%
ALDURAZYME
57 54 6% 137 159 (14)%
POMBILITI + OPFOLDA 49 — NM 79 — NM
Total Metabolic Conditions Revenue $744 $516 44% $1,954 $1,556 26%
KUVAN
$23 $24 (4)% $71 $76 (7)%
ROCTAVIAN(1)
$1 $3 (67)% $15 $23 (35)%
GAAP Net Income (Loss) $(123) $(31) 297% $27 $395 (93)%
Non-GAAP Income (2)
$45 $22 105% $430 $525 (18)%
GAAP Operating Margin % (3)
(8.0)% (6.0)% 5.7% 19.4%
Non-GAAP Operating Margin % (2)
13.4% 2.8% 24.6% 26.3%
GAAP Diluted EPS
$(0.63) $(0.16) 294% $0.14 $2.04 (93)%
Non-GAAP Diluted EPS (2)
$0.23 $0.12 92% $2.18 $2.69 (19)%

(Press release, BioMarin, NOV 2, 2026, View Source [SID1234674029])