BioMarin’s Voxzogo met the primary endpoint in the Phase III CANOPY‑HCH‑3 trial, producing a 2.33 cm greater annual height velocity versus placebo at 52 weeks. The study also showed a 2.35 cm gain in standing height and arm span and improvements in height Z‑score, with no serious treatment‑related adverse events reported. BioMarin has filed an NDA with the FDA; approval would make Voxzogo the first targeted therapy for hypochondroplasia in the US (estimated 8,500–23,000 people).
Voxzogo Delivers Statistically Significant Height Gains in Phase III Hypochondroplasia Trial
BioMarin has filed for Voxzogo's US approval in hypochondroplasia
BioMarin has reported updated Phase III results showing that its investigational therapy Voxzogo (vosoritide) produced meaningful growth benefits for children with hypochondroplasia, meeting the trial's primary endpoint.
Key Results
Data from the late-stage CANOPY‑HCH‑3 study (NCT06455059), presented at the 2026 European Society for Paediatric Endocrinology (ESPE) meeting, showed that children treated with Voxzogo achieved a statistically significant 2.33 cm greater annual height velocity versus placebo after 52 weeks. The study also recorded a 2.35 cm increase in standing height and arm span, along with improvements in height Z‑score compared with controls.
Safety and Patient Impact
Voxzogo preserved a consistent safety profile across the trial population. Investigators reported no serious adverse events attributed to treatment in this study. In addition to objective growth measures, the trial showed numerical improvements in patient‑reported quality of life measures.
Regulatory Next Steps and Potential Impact
Greg Friberg, BioMarin's EVP and Chief R&D Officer, described the dataset as providing "a comprehensive picture" of Voxzogo's effects on multiple aspects of growth in children with hypochondroplasia, a rare genetic disorder characterized by underdevelopment of the long bones. BioMarin has submitted a New Drug Application (NDA) to the US Food and Drug Administration (FDA) seeking approval of Voxzogo for hypochondroplasia.
If cleared by the FDA, Voxzogo would be the first targeted therapy available for hypochondroplasia in the United States. BioMarin estimates the US population affected by hypochondroplasia to be roughly 8,500–23,000 people. Approval would also broaden Voxzogo’s role beyond its current indication for patients with achondroplasia who have open growth plates.
Study: CANOPY‑HCH‑3 (NCT06455059). Data Presented: ESPE 2026.
Notes: All efficacy and safety findings described are from the 52‑week data cut; longer follow‑up and regulatory review will determine next clinical and commercial steps.
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