Biomarin Receives FDA Accelerated Approval for Voxzogo (vosoritide) for Treating Children with Achondroplasia, a Rare Genetic Disorder

Biomarin announced the U.S FDA has granted accelerated approval to Voxzogo (vosoritide) for Injection, indicated to increase linear growth in pediatric patients with achondroplasia five years of age and older with open epiphyses (growth plates).  This indication is approved under accelerated approval based on an improvement in annualized growth velocity (AGV). Full approval is contingent upon the company verifying clinical benefit in confirmatory studies.  To fulfill this post-marketing requirement, BioMarin intends to use the ongoing open-label extension studies compared to available natural history.


