Daily vosoritide injections boosted growth in hypochondroplasia trial
Eighty-one children with hypochondroplasia, aged 3 to less than 18 years, received either daily subcutaneous vosoritide or placebo for 52 weeks in a phase 3 trial. Growth velocity increased by a least squares mean of 1.95 cm per year in the vosoritide arm versus -0.39 cm per year with placebo. That translates to a difference of 2.33 cm per year (95% confidence interval, 1.85 to 2.82 cm per year; two-sided P less than 0.001), according to results published in 2026. BioMarin Pharmaceutical funded the study. Hypochondroplasia is caused by FGFR3 mutations and produces disproportionate short stature. Vosoritide is a C-type natriuretic peptide analogue already approved for achondroplasia.
No targeted therapies exist for hypochondroplasia, a skeletal condition that causes short stature in children. This trial shows a drug can significantly increase linear growth over one year.
Written by the Genomes desk from the primary source cited and linked above and checked against it. Research use only; not medical advice. Corrections: [email protected].