Posted by Michael Wonder on 24 Jul 2020
BioMarin submits marketing authorisation application to European Medicines Agency for vosoritide to treat children with achondroplasia
23 July 2020 - If approved, vosoritide would be the first medicine to treat Achondroplasia in EU.
BioMarin Pharmaceutical announced today that the company submitted a marketing authorisation application to the EMA for vosoritide, an investigational, once daily injection analog of C-type natriuretic peptide for children with achondroplasia, the most common form of disproportionate short stature in humans. Subject to completion of EMA's validation check, BioMarin anticipates the start of the review of the application to commence in August 2020.
The marketing application is based on the outcomes from the randomised, double-blind, placebo-controlled Phase 3 study evaluating the efficacy and safety of vosoritide, announced in December 2019, and further supported by the long-term safety and efficacy from the ongoing Phase 2 and Phase 3 extension studies, and extensive natural history data. If approved, vosoritide would be the first medicine for the treatment of achondroplasia in Europe.
