BioMarin raises 2026 revenue guidance after Amicus acquisition and FDA PALYNZIQ expansion

BioMarin Pharmaceutical Inc. reported first quarter 2026 total revenues of $766 million and increased full-year 2026 total revenues guidance to between $3.825 billion and $3.925 billion. The accelerated growth rate of 20% Y/Y at the midpoint reflects the addition of GALAFOLD® and POMBILITI® + OPFOLDA® to BioMarin's portfolio following the completion of the Amicus Therapeutics acquisition.

In February, U.S. FDA approved PALYNZIQ® for adolescents 12 years of age and older with phenylketonuria (PKU); EU approval for adolescents 12 years of age and older is expected in 2026. In April, the company submitted its U.S. supplemental new drug application (sNDA) for full approval of VOXZOGO® for achondroplasia, and expects to be notified of sNDA acceptance by Q3 2026.

In the second quarter, BioMarin expects to share BMN 401 Phase 3 topline data in children ages 1-to-12 year-old with ENPP1 deficiency. Regulatory submissions are anticipated in 2H'26 should the data be supportive, with a potential first-in-disease launch in 2027. In the second quarter, the company also expects to share Phase 3 topline data for VOXZOGO for hypochondroplasia; regulatory submissions are anticipated in 2H'26 should the data be supportive, with a potential first-in-class launch in 2027.

In March, the company presented initial Phase 1/2 data for BMN 351 demonstrating dose-dependent increases in dystrophin expression at Week 25 biopsy in both the 6 and 9 mg/kg dose cohorts. Clinical biomarkers, including decreases in creatine kinase, suggested improvements in overall muscle health beyond the Week 25 time point, and longer-term outcomes from both NSAA and 6MWT suggested a prevention of functional decline when compared to historical matched controls. The 12 mg/kg dose cohort continues to enroll, with a data update expected by year-end.

In April, the first patient was enrolled in the registration-enabling Phase 2/3 study of BMN 333, BioMarin's long-acting C-type natriuretic peptide (CNP) for achondroplasia. A data update from this study is expected in 2027. In May, BioMarin reported new data demonstrating the benefits of long-term treatment with VOXZOGO, including improvements in arm span, bone health, and quality of life. Data from ongoing long-term extension clinical trials showed that children who initiated VOXZOGO treatment after age 5 achieved mean height gains of +10.60cm after six years and +13.59cm (p<0.0001 for both) after eight years of treatment, as compared to natural history data.

Enzyme Therapies revenue grew 6% Y/Y in the first quarter, driven by revenue growth for VIMIZIM®, NAGLAZYME®, and BRINEURA®. Continued underlying patient demand in Q1 for PALYNZIQ was driven by an increase in enrollments and new starts in the under-18-year age group following label expansion in February, and full-year 2026 PALYNZIQ revenue is expected to increase year-over-year. The number of children being treated with VOXZOGO increased by more than 20% Y/Y in the first quarter, while large VOXZOGO orders in fourth quarter of 2025 resulted in modest Y/Y growth of 3% in the first quarter of 2026.