Description

Achondroplasia inhibits endochondral ossification. Vosoritide is a biologic analogue of C-type natriuretic peptide – a potent stimulator of endochondral ossification. Animal models have shown that once-daily vosoritide promotes long-bone growth, and corrects the dwarfism phenotype in mice with achondroplasia.

This multinational, Phase 2, dose-finding and extension study evaluated the safety and side-effect profile of four doses of vosoritide in 35 children with achondroplasia. In the first 6 months, a dose-dependent increase in annualised growth velocity was observed, with no adverse events related to disproportionate skeletal growth, and no deaths or clinically significant adverse cardiovascular effects. Since no difference in efficacy or safety was identified between the 15.0 and 30.0 μg/kg doses, the lower dose was progressed in further studies.