Results
8Second Quarter 2026 Total Revenues Increased 20% Year-over-year to $990 million
Outlook
Stronger Growth Expectations Drive Increased Guidance for Full-year 2026 Total Revenues, VOXZOGO ® , and Non-GAAP Diluted Earnings Per Share (EPS)Guidance raised
Addition of GALAFOLD ® and POMBILITI ® + OPFOLDA ® , with Cost Synergies, Expected to Accelerate Revenue Growth, Non-GAAP Diluted EPS Accretion, Non-GAAP Operating Margin Expansion, and Operating Cash Flow through the Mid-2030s
Management commentary
"This quarter, we executed strongly across our portfolio while rapidly integrating Amicus into BioMarin's operations and advancing plans to accelerate growth for GALAFOLD and POMBILITI + OPFOLDA, and extending the benefit of these medicines to more patients worldwide," said Alexander Hardy, President and Chief Executive Officer of BioMarin.
1“Strong global demand led us to increase full-year VOXZOGO revenue guidance to at least $1 billion in 2026.Guidance raised
Adding to this momentum is the opportunity to advance our second potential indication with VOXZOGO, hypochondroplasia, based on recent pivotal data that exceeded our expectations.” Mr. Hardy added, “With our larger, more diversified commercial portfolio of innovative medicines, we are positioned to deliver additional growth and increased profitability.
We expect strong execution through the remainder of 2026, bringing together our expanded portfolio, scale and disciplined integration efforts to reach more patients living with serious genetic conditions around the world.”
2026 Business and Pipeline Highlights
Innovation
Corporate events
BioMarin recently submitted its supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for the approval of VOXZOGO for the treatment of hypochondroplasia.
If approved, VOXZOGO would be the first targeted therapy for the treatment of hypochondroplasia, with a potential 2027 launch.
The company plans to provide an update on the application status as part of its third quarter earnings update.
Results
In May, the company announced that the Phase 3 CANOPY-HCH-3 study of VOXZOGO in children with hypochondroplasia met its primary endpoint, with a statistically significant increase in annualized growth velocity (AGV) at week 52 versus placebo (LS mean difference +2.33 cm/yr, p<0.0001), along with significant improvements in standing height, height Z-score, and the key secondary endpoint of arm span.
The full Phase 3 dataset will be shared in a late-breaking oral presentation at the European Society for Paediatric Endocrinology Annual Meeting in September.
In June, at the Endocrine Society Annual Meeting (ENDO 2026), a Phase 2 investigator-sponsored three-year extension study of VOXZOGO in 13 children with hypochondroplasia showed sustained improvements in growth with a favorable safety profile.
Mean AGV increased from 4.27 cm/year at baseline to 7.24 cm/year at year one (p<0.001) and remained above baseline through year three, with mean height standard deviation score (SDS) improving 0.72 over the three years.
Corporate events
Also at ENDO 2026, the company presented Phase 1 data for BMN 333, BioMarin's long-acting C-type natriuretic peptide (CNP) for achondroplasia.
In a single-ascending-dose study in healthy adults, BMN 333 demonstrated sustained exposure supporting weekly dosing and was well tolerated, with free CNP exposure at the highest dose more than 13-fold that of another long-acting CNP agent, reflecting its potential to become a new standard of care in achondroplasia.
The Phase 2/3 study is enrolling, with a data update expected in 2027.
In July, BioMarin announced that the FDA accepted its sNDA for full approval of VOXZOGO in children with achondroplasia, with a Prescription Drug User Fee Act (PDUFA) target action date of February 28, 2027.
In the second quarter, the European Commission approved PALYNZIQ ® for adolescents 12 years and older with phenylketonuria (PKU).
PALYNZIQ is the only therapy that enables people with PKU to reach physiologic Phe levels while reducing dietary restrictions, regardless of severity.
During the quarter, BioMarin added BMN 820 (formerly DMX-200) to its portfolio, a first-in-class oral CCR2 inhibitor for focal segmental glomerulosclerosis (FSGS) for which BioMarin holds exclusive U.S. commercialization rights.
BMN 820 has the potential to treat a broad FSGS population, regardless of nephrotic syndrome status, and represents a U.S. total addressable patient population of approximately 30,000.
The Phase 3 ACTION 3 trial is ongoing, with pivotal data expected in 2028.
Outlook
BMN 351, BioMarin's Phase 1/2 candidate for Duchenne muscular dystrophy, continued in development.
The company expects to provide a program update by year-end.
Corporate events
Following the pivotal ENERGY 3 trial results, previously announced in May, in which BMN 401 did not meet one of its two co-primary endpoints for the treatment of ENPP1 deficiency, BioMarin has now made the decision to discontinue development of BMN 401 across all indications.
In July, BioMarin and the n-Lorem Foundation entered a collaboration and global exclusive license agreement to develop a first-in-disease antisense oligonucleotide (ASO) medicine for ReNU syndrome, a serious, rare neurodevelopmental condition with no approved targeted therapies.
ReNU syndrome has an expected global population of approximately 100,000.
Management commentary
Growth
Outlook
BioMarin expects peak revenue for GALAFOLD to be approximately $1.4 billion by the mid-2030s and for POMBILITI + OPFOLDA to be approximately $1.2 billion by the mid-to-late-2030s.
BioMarin expects these high growth therapies to benefit from its global scale and proven commercial capabilities.
Results
Metabolic Conditions (formerly Enzyme Therapies) revenue grew 25% Y/Y in the second quarter of 2026, driven by the additions of GALAFOLD and POMBILITI + OPFOLDA and continued strength from PALYNZIQ.
The number of patients on therapy grew across all BioMarin-marketed therapies, both Y/Y and sequentially.
Outlook
2Strong U.S. and global demand led to increased full-year 2026 VOXZOGO revenue guidance to a low end of $1 billion.Guidance raised
The number of children being treated with VOXZOGO globally increased by more than 20% Y/Y in the second quarter.
In the U.S., the majority of new patient starts were under two years of age, and the region drove approximately 25% of total VOXZOGO revenue during the quarter.
Management commentary
Value Commitment
Outlook
7As part of the acquisition of Amicus, which closed on April 27, 2026, the company identified 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.
Synergies reflect a reduction of Amicus’ legacy labor costs and external spend and are expected to be largely driven by general and administrative functions, with the large majority of sales and marketing capabilities retained to support continued commercial growth.
GALAFOLD and POMBILITI + OPFOLDA, combined, are expected to reach over 60% Non-GAAP Operating Margin by 2030.
3The company is targeting gross leverage below 2.5 times by mid-year 2027, an acceleration by approximately one year of prior timeline guidance provided at deal announcement, supported by profitability growth of the combined company.Guidance raised
Results
Second Quarter 2026 Financial Highlights
Total Revenues for the second quarter of 2026 were $990 million, an increase of $165 million compared to the same period in 2025, primarily driven by revenues from GALAFOLD and POMBILITI + OPFOLDA, which were acquired from Amicus on April 27, 2026, as well as new patients initiating VOXZOGO therapy across all regions and growth in U.S. patients treated with PALYNZIQ.
These increases were partially offset by lower VIMIZIM ® revenue due to the timing of large government orders outside the U.S. and lower ALDURAZYME ® sales volume due to the timing of order fulfillment to Sanofi.
5GAAP Net Income for the second quarter of 2026 decreased to $45 million compared to $241 million for the same period in 2025.
The decrease was primarily driven by the acquisition of Amicus, including integration and restructuring costs, intangible asset amortization, interest expense from debt issued to finance a portion of the transaction, and amortization of inventory fair value step-up.
Other drivers included higher sales and marketing spend to support newly acquired products and global expansion of VOXZOGO and higher Research and Development (R&D) spend related to BMN 401, which was acquired in the third quarter of 2025, partially offset by higher gross profit driven by revenue growth as described above.
6Non-GAAP Income for the second quarter of 2026 decreased to $236 million compared to $282 million for the same period in 2025.
The decrease was primarily driven by higher interest expense, higher sales and marketing spend to support newly acquired products and global expansion of VOXZOGO, and higher R&D spend related to BMN 401, partially offset by higher gross profit driven by revenue growth as described above.
Corporate events
(1) In 2026, the company announced that it will no longer market ROCTAVIAN.
Outlook
Updated 2026 Full-Year Financial Guidance (in millions, except EPS amounts)
4Total 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.Guidance raised
Guidance reflects post-close contributions from Amicus beginning April 27, 2026.
(3) Non-GAAP Diluted EPS guidance assumes approximately 200 million Weighted-Average Diluted Shares Outstanding.
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