San Rafael, Calif. – September 13, 2026 -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) reported that its Phase 3 CANOPY-HCH-3 trial of VOXZOGO (vosoritide) met its primary endpoint in children with hypochondroplasia, with results published in NEJM Evidence and presented at the European Society for Paediatric Endocrinology 2026 Annual Meeting.
Trial data show statistically significant gains across four growth metrics
After 52 weeks, VOXZOGO produced a least squares mean difference of 2.33 cm/year in annualized growth velocity versus placebo (p0.0001). Standing height improved by 2.35 cm (p0.0001), height Z-score by 0.39 standard deviation score (p0.0001), and arm span by 1.03 cm (p=0.0082). Children on VOXZOGO also showed numerical improvements in quality-of-life measures, with longer-term follow-up ongoing. BioMarin said the safety profile matched prior VOXZOGO studies in achondroplasia, with most adverse events mild and no treatment-related serious adverse events reported.
BioMarin files sNDA to become first approved treatment for the condition
BioMarin has submitted a supplemental New Drug Application to the FDA seeking approval of VOXZOGO for hypochondroplasia, with submissions to the European Medicines Agency and other regional regulators on track. No medicine is currently approved by the FDA or EMA for hypochondroplasia. If cleared, VOXZOGO would become the first targeted therapy for the condition, with BioMarin targeting a 2027 launch.
Company estimates 14,000 children could be eligible for treatment
BioMarin estimates roughly 14,000 children with hypochondroplasia within its global footprint may be eligible for VOXZOGO if approved. Greg Friberg, Executive Vice President and Chief Research & Development Officer at BioMarin, said the data package supports the company's goal of securing approval for the first medicine developed for children with hypochondroplasia. Andrew Dauber, lead study investigator and Chief of Endocrinology at Children's National in Washington, D.C., said the observed changes in growth velocity and arm span reinforce the drug's potential as a targeted option for the condition.
VOXZOGO already treats over 5,000 children with achondroplasia in 50-plus countries
VOXZOGO, first approved in 2021, is currently indicated for achondroplasia in the U.S., Japan, Australia and the EU. A separate sNDA covering long-term safety and efficacy data, including adult height outcomes, is under FDA review with a PDUFA target action date of Feb. 28, 2027.