Detailed narrative
Amicus Integration & Growth Strategy
BioMarin completed the Amicus acquisition, adding Galafold for Fabry disease and Pombiliti/Opfolda for Pompe disease to its commercial portfolio. The integration plan focuses on leveraging BioMarin's operating scale and capabilities to drive diagnosis and treatment rates for these conditions. The company plans to share a detailed roadmap, including commercial and cost synergy outlook, on the next quarterly call. Management reiterated previous peak revenue potential estimates of approximately $1 billion each for Galafold and Pombiliti/Opfolda, with potential for these figures to be higher.
VOXZOGO Momentum & Expansion
VOXZOGO continues to demonstrate strong patient demand, with new patient starts increasing across all regions. In the U.S., over half of new patient starts in Q1 were from children under age 2, and the average age of initiation in this segment decreased by 10%. The sNDA for full approval of VOXZOGO has been submitted to the FDA. The company is also preparing for potential expansion into hypochondroplasia, with Phase III data expected in Q2 2026 and potential approval in 2027, supported by pre-commercialization activities focused on diagnosis and awareness.
Enzyme Therapies Performance
The enzyme therapies portfolio delivered 6% year-over-year growth in Q1, led by VIMIZIM, NAGLAZYME, and BRINEURA. PALYNZIQ saw encouraging early momentum following its adolescent label expansion in the U.S. in February, with meaningful enrollments and new patient starts in the under-18 age group. The company expects year-over-year revenue growth for PALYNZIQ in full year 2026 as these new patient starts gain momentum and patients titrate to their maintenance dose.
Pipeline Progress & Milestones
BioMarin anticipates several key R&D milestones in 2026, including Phase III top-line results for VOXZOGO in hypochondroplasia and BMN 401 in ENPP1 deficiency, both expected in Q2. Enrollment is underway for the global registrational-enabling Phase II/III study of BMN 333, a long-acting CNP therapy for achondroplasia, aiming for a superiority profile. Initial data for BMN 351 in muscular dystrophy showed dose-dependent dystrophin increases and functional improvements, with enrollment ongoing in the 12 mg/kg cohort.
Financial Outlook & Timing
Q1 2026 total revenues were $766 million, with non-GAAP diluted EPS of $0.76. These results were impacted by a $31 million charge for an unsuccessful NAGLAZYME manufacturing process qualification and pre-close Amicus acquisition costs. The company expects more than 55% of total 2026 revenues and approximately two-thirds of 2026 EPS to be recognized in the second half of the year, driven by order timing for historical products and the weighting of Amicus revenues and dilution.
Hypochondroplasia Market Preparation
BioMarin is actively engaged in pre-launch activities for VOXZOGO in hypochondroplasia, focusing on driving diagnosis and awareness. These non-promotional efforts have already led to a 90% increase in identified hypochondroplasia patients and a 70% reduction in the average age of diagnosis. The goal is to ensure patients are identified and ready for therapy upon potential launch, accelerating the adoption curve.