BioMarin Reports Positive VOXZOGO Phase 3 Results and Q4 2025 Revenue Beat
Positive Phase 3 VOXZOGO results in hypochondroplasia; FDA sNDA submission planned in Q3. Q4 2025 revenue rose 17% to $874.6M, beating estimates; EPS missed. Q1 2026 results due May 4.
BioMarin Pharmaceutical reported positive Phase 3 Pivotal study results for VOXZOGO in children with hypochondroplasia, with the study meeting its primary endpoint and showing a highly statistically significant improvement in annualized growth velocity (AGV). The company also released fourth-quarter 2025 earnings on May 13, 2026, with revenue of $874.6 million, up 17% year over year and beating analyst estimates of $834.3 million by 4.8%.
BioMarin intends to submit a supplemental New Drug Application to the US Food and Drug Administration in the third quarter and plans to make submissions to the European Medicines Agency. Hypochondroplasia is a rare genetic disorder characterized by impaired bone growth, resulting in disproportionate short stature and skeletal abnormalities. For the first time, a Phase 3 study of hypochondroplasia has demonstrated impressive gains in growth, including improvements in arm span. Safety findings were in line with the established profile in achondroplasia.
Adjusted EPS came in at $0.46, 17.9% below the $0.56 consensus. The stock traded down 2.7% to $61.21 immediately after the report. Voxzogo, approved for achondroplasia, drove significant growth in Q4 2025, contributing to the 17% revenue increase to $874.6 million for the period ended December 31, 2025. Legacy products like Vimizim and Naglazyme provide stable cash flows. Full-year 2025 revenue hit around $3.38 billion, aligning with estimates. Guidance for 2026 adjusted EPS at $5.05 midpoint trails consensus slightly.
BioMarin will host a conference call and webcast on Monday, May 4, 2026, at 4:30 p.m. ET to discuss first quarter 2026 financial results and provide a general business update, led by the company's President and Chief Executive Officer. Market expectations call for first-quarter revenue to grow 4.1% year on year, slowing from the 14.8% increase recorded in the same quarter last year. The company has missed Wall Street's revenue estimates multiple times over the last two years.
BioMarin, founded in 1997 and based in San Rafael, California, is a global rare disease biotechnology company with eight commercial therapies and a strong clinical and preclinical pipeline. It is a pioneer in enzyme replacement therapies and gene therapy.