VOXZOGO

May 4, 2026: BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) reported Q1 2026 results and outlined multiple upcoming pipeline/regulatory catalysts following completion of the Amicus Therapeutics acquisition (adding GALAFOLD for Fabry disease and POMBILITI + OPFOLDA for Pompe disease) and raised 2026 total revenue guidance to $3.825–$3.925B.

Key catalyst and program updates:

• PALYNZIQ (PKU): U.S. FDA approved label expansion in February for adolescents age ≥12; EU approval for adolescents is expected in 2026.
• BMN 351 (Duchenne muscular dystrophy): Initial Phase 1/2 data presented in March at the MDA Clinical & Scientific Congress showed dose-dependent dystrophin increases at Week 25 (6 and 9 mg/kg) plus biomarker/functional signals; 12 mg/kg cohort enrolling; data update expected by year-end.
• BMN 333 (achondroplasia): First patient enrolled in April in a registration-enabling Phase 2/3; data update expected in 2027.
• VOXZOGO (achondroplasia): U.S. sNDA submitted in April for full approval; acceptance notification expected by Q3 2026. New long-term extension data were reported in May at the Pediatric Endocrine Society meeting.
• BMN 401 (ENPP1 deficiency): Phase 3 topline data expected in Q2 2026; regulatory submissions anticipated in 2H 2026 if supportive; potential launch in 2027.
• VOXZOGO (hypochondroplasia): Phase 3 topline data expected in Q2 2026; regulatory submissions anticipated in 2H 2026 if supportive; potential launch in 2027.