P131: Persistence of growth-promoting effects in infants and toddlers with achondroplasia: Results from a phase II extension study with vosoritide*

This is the authors' abstract. We don't add key points for this paper.

Vosoritide, a potent stimulator of endochondral bone growth, is approved for use in children with achondroplasia (ACH) until closure of epiphyses. We recently reported results from a phase 2, randomized, double-blind, placebo-controlled study (111-206) of vosoritide in young children with achondroplasia aged 3 months – <5 years. Here we report safety and efficacy results from the open-label extension study (111-208) spanning up to 4 years of treatment in children aged <5 years at the start of vosoritide therapy.