Phase 3 study shows vosoritide improves growth in children with hypochondroplasia - Children's National

A Phase 3 multicenter study, published in NEJM Evidence and presented at the 64th Annual Meeting of the European Society for Paediatric Endocrinology (ESPE), found that vosoritide may increase growth in children with hypochondroplasia, a rare genetic skeletal condition with limited treatment options.

Andrew Dauber, MD, MMSc, chief of Endocrinology and director of the Center for Precision Medicine and Genomics Research at Children's National, led the BioMarin-sponsored CANOPY-HCH-3 trial. The global study enrolled 81 children ages 3 to 17 with genetically confirmed hypochondroplasia and randomly assigned them to receive vosoritide or a placebo.

Why it matters

After 52 weeks, children treated daily with vosoritide had a 2.33 cm per year greater improvement in annualized growth velocity than those who received a placebo. Researchers also reported significant improvements in standing height, height Z-score and arm span, with no new safety signals.

"Hypochondroplasia can affect a child's growth, physical function, and everyday life, with families often navigating unique challenges as they support their child's development," said Dr. Dauber. "The improvements 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."

Dr. Dauber delivered the oral presentation at the 2026 meeting in Marseille, France. Participants will continue to be followed in a long-term extension study.

Leading the way

Researchers at Children's National led the first ever trial of vosoritide for short stature not caused by achondroplasia. The Phase 2 basket trial found increased growth in children with hypochondroplasia and three other rare genetic growth disorders - RASopathies, ACAN mutations and NPR2 deficiency, while the world's first vosoritide trial for Turner syndrome reported encouraging early growth results.

Read the full study, A Phase 3 Trial of Vosoritide in Children with Hypochondroplasia, in NEJM Evidence.