Andrew Dauber, MD, Chief of Endocrinology at Children’s National Hospital, discusses results from a phase 3 study of Voxzogo (vosoritide) in children with hypochondroplasia.
Hypochondroplasia is a rare skeletal disease characterized by very short stature, similar to achondroplasia, but with milder features. People with hypochondroplasia usually have very short stature, large head, accentuated lordosis, short arms and legs, and broad, short hands and feet. Other features include a limited range of motion in the elbows, lordosis, and bowed legs. Uncommon symptoms may include learning difficulties and convulsions. Hypochondroplasia is often caused by genetic changes in the FGFR3 gene.
The CANOPY-HCH-3 (NCT06455059) clinical trial is a phase 3, double-blind, placebo-controlled, multicenter, open-label, long-term extension study evaluating the safety and efficacy of vosoritide in children with hypochondroplasia. Vosoritide is a C-type natriuretic peptide (CNP) analog approved by the US Food and Drug Administration (FDA) to promote linear bone growth in children with achondroplasia.
The trial met its primary endpoint, with vosoritide demonstrating a statistically significant increase in the change from baseline at week 52 in annualized growth velocity (AGV) compared to placebo. Treatment with vosoritide also showed a statistically significant increase in standing height and height Z-score versus placebo after one year of treatment. Additionally, significant improvements in arm span were observed.
The safety profile of vosoritide was consistent with the established profile in achondroplasia and no new safety signals were observed.
A supplemental New Drug Application (sNDA) submission to the FDA is planned for the third quarter of 2026.
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To learn more about hypochondroplasia and other rare musculoskeletal conditions, visit https://checkrare.com/diseases/musculoskeletal-diseases/