Detailed Phase 3 CANOPY-HCH-3 data in children living with hypochondroplasia also featured in a late-breaking oral presentation at the European Society for Paediatric Endocrinology (ESPE) 2026 Annual Meeting

VOXZOGO demonstrated statistically significant improvements across multiple measures of growth, including annualized growth velocity, standing height, height Z-score and arm span

BioMarin recently submitted a supplemental New Drug Application (sNDA) to the FDA to support expanding treatment with VOXZOGO to include children with hypochondroplasia

BioMarin Pharmaceutical Inc. (Nasdaq: BMRN)  announced new data from the Phase 3 CANOPY-HCH-3 study evaluating VOXZOGO^® (vosoritide) in children with hypochondroplasia were published in New England Journal of Medicine (NEJM) Evidence and presented at the European Society for Paediatric Endocrinology (ESPE) 2026 Annual Meeting. The data included new results on the magnitude of benefit seen in children receiving VOXZOGO, including statistically significant improvements in annualized growth velocity (AGV), standing height, height Z-score and arm span after 52 weeks, with safety findings consistent with the established profile of VOXZOGO in achondroplasia.

The CANOPY-HCH-3 study showed that treatment with VOXZOGO led to a statistically significant improvement in AGV compared with placebo after 52 weeks, meeting the study's primary endpoint (least squares [LS] mean difference of 2.33 cm/year; p<0.0001). Children treated with VOXZOGO also showed significant improvements in standing height (LS mean difference of 2.35 cm; p<0.0001), height Z-score (LS mean difference of 0.39 standard deviation score; p<0.0001), and arm span (LS mean difference of 1.03 cm; p=0.0082) compared with placebo. Children who received VOXZOGO also demonstrated numerical improvements in quality of life, and follow-up will continue to assess the impact of treatment over a longer term. The overall safety profile was consistent with previous studies of VOXZOGO, with most adverse events reported as mild and no treatment-related serious adverse events identified.

"These results presented in detail for the first time provide a comprehensive picture of the impact of VOXZOGO across multiple measures of growth in children with hypochondroplasia," said Greg Friberg, M.D., Executive Vice President and Chief Research & Development Officer at BioMarin. "Based on this compelling body of evidence, we have submitted these data to the FDA with the goal of securing approval for the first medicine for children with hypochondroplasia."

"Hypochondroplasia can affect a child's growth, physical function and everyday life, with families often navigating unique challenges as they support their children's development," said Andrew Dauber, M.D., lead study investigator and Chief of Endocrinology at Children's National in Washington, D.C. "The changes we observed in annualized growth velocity and arm span provide encouraging evidence of how children with hypochondroplasia responded to treatment throughout the study. These findings deepen our understanding of the condition while reinforcing VOXZOGO's potential as the first targeted medicine developed specifically for children with hypochondroplasia."

BioMarin recently submitted its supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for the approval of VOXZOGO for the treatment of hypochondroplasia and are on track with the submissions to the European Medicines Agency (EMA) and other regional health authorities. If approved, VOXZOGO would be the first targeted therapy for the treatment of hypochondroplasia, with a potential 2027 launch.