Vosoritide speeds growth in children with hypochondroplasia in phase 3 trial

NEJM Evidence published CANOPY-HCH-3, a phase 3 trial of vosoritide (Voxzogo), a C-type natriuretic peptide analogue already approved for achondroplasia, in 81 children with hypochondroplasia, a rare genetic skeletal dysplasia that causes short stature. After 52 weeks, annualised growth velocity rose by 1.95 cm per year with vosoritide and fell by 0.39 cm per year with placebo, a difference of 2.33 cm per year, with no grade 3 or higher adverse events. BioMarin has applied for US approval, with an FDA decision expected by February 28, 2027; no medicine is currently approved by the FDA or EMA for hypochondroplasia.