BioMarin Reports Positive Phase 3 VOXZOGO Data

San Rafael, California, September 9, 2026

Phase 3 Study Meets Primary Growth Endpoint

The global CANOPY-HCH-3 Phase 3 study evaluated VOXZOGO in children with hypochondroplasia and compared treatment outcomes with placebo over 52 weeks. The study met its primary endpoint, demonstrating a statistically significant improvement in annualized growth velocity (AGV). Children receiving VOXZOGO achieved a least-squares mean improvement of 2.33 centimeters per year compared with placebo, with a statistical significance of p<0.0001. Additional growth measures also favored VOXZOGO. At 52 weeks, treatment was associated with a 2.35-centimeter improvement in standing height compared with placebo, while the height Z-score improved by 0.39 standard deviation score. The study also reported a statistically significant 1.03-centimeter improvement in arm span, an outcome that may have functional relevance for children living with skeletal dysplasia. The arm-span result reached statistical significance with p=0.0082. The findings provide a broader assessment of treatment effects than growth velocity alone, with BioMarin highlighting changes in standing height, height Z-score and arm span as additional measures of the potential clinical benefit of vosoritide in hypochondroplasia.

Safety Profile Remains Consistent

The safety findings from CANOPY-HCH-3 were consistent with the established safety profile of VOXZOGO observed in its development for achondroplasia. According to BioMarin, most adverse events reported in the Phase 3 study were mild, and there were no treatment-related serious adverse events identified. Follow-up will continue to assess longer-term outcomes, including the potential impact of treatment on growth and quality of life. Hypochondroplasia is a rare genetic skeletal dysplasia characterized by impaired bone growth and disproportionate short stature. The condition can affect the long bones, spine and other skeletal structures and may have consequences for physical functioning and quality of life. BioMarin estimates that approximately 14,000 children within its global footprint could potentially be eligible for treatment with VOXZOGO if the therapy receives approval for this indication. Currently, there are no medicines approved by the FDA or European Medicines Agency specifically for hypochondroplasia, making the development of targeted treatment options an area of significant unmet medical need.

FDA Submission Supports Potential New Indication

BioMarin has now submitted a supplemental New Drug Application to the FDA seeking approval of VOXZOGO for children with hypochondroplasia. The company is also progressing planned submissions to the European Medicines Agency and other regional health authorities. If approved, BioMarin states that VOXZOGO could become the first targeted therapy for hypochondroplasia, with a potential launch in 2027. VOXZOGO is already an approved medicine for increasing linear growth in children with achondroplasia and open growth plates in several markets, including the United States, Japan and Australia, with an indication in the European Union for children with achondroplasia aged four months and older whose growth plates have not closed. Its mechanism involves acting as a C-type natriuretic peptide (CNP) analog to promote endochondral bone growth downstream of FGFR3 signaling. Importantly, VOXZOGO has not yet been approved for hypochondroplasia by any regulatory agency. The positive Phase 3 findings and subsequent regulatory submissions therefore represent an important step in BioMarin’s effort to expand the therapeutic application of vosoritide to another rare skeletal disorder. Continued regulatory review and longer-term clinical evidence will determine whether the data support approval for this potential new indication.

Source: BioMarin press release