BioMarin’s VOXZOGO® meets Phase 3 endpoints in hypochondroplasia, showing significant growth gains and functional improvements with strong safety.
Written By: Fariha Sameen, PharmD
Reviewed By: Pharmacally Editorial Team
BioMarin Pharmaceutical reported positive pivotal results from the Phase 3 CANOPY-HCH-3 trial (Study 111-303; NCT06455059), showing that VOXZOGO® (vosoritide) significantly improved growth outcomes in children with hypochondroplasia, a rare FGFR3-related skeletal dysplasia with no approved therapies.
VOXZOGO is a C-type natriuretic peptide analog designed to counter overactive FGFR3 signaling, the underlying molecular driver of the condition.
Over 52 weeks, children treated with VOXZOGO achieved least squares mean increase in annualized growth velocity (AGV) of +2.33 cm/year versus placebo (p < 0.0001), meeting the study’s primary endpoint. The therapy also produced statistically significant improvements in standing height and height Z-scores (p < 0.0001).
Importantly, the study demonstrated significant gains in arm span versus placebo (p = 0.004), a functional measure that may support improved reach and activities of daily living for affected children.
“These results represent a long-awaited milestone for children with hypochondroplasia and their families,” said Dr. Andrew Dauber, Chief of Endocrinology at Children’s National Hospital and the study’s lead investigator.
BioMarin Chief Research & Development Officer Greg Friberg, M.D., said the results exceeded company expectations, particularly the improvements observed across both growth velocity and body proportionality measures.
The global randomized, placebo-controlled trial enrolled 80 children between 3 and 17 years of age. VOXZOGO’s safety profile was consistent with prior achondroplasia studies, with no new safety signals identified.
The findings build on earlier data reported by BioMarin Pharmaceutical in March 2026 showing that early treatment with VOXZOGO improved growth, body proportionality, and skeletal development in children with achondroplasia.
About the Writer
Fariha Sameen, PharmD (LinkedIn), is a clinical pharmacy professional with hands-on experience in patient counselling, medication review, therapeutic monitoring, and clinical documentation across multiple departments. She has experience identifying and assessing drug-related problems and supporting medication safety practices. Her interests include pharmacovigilance, ADR reporting, clinical research, and medical writing focused on clear, evidence-based communication.