BioMarin Pharmaceutical Inc. announced that the company submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for vosoritide, an investigational, once daily injection analog of C-type Natriuretic Peptide (CNP) 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 MAA review to commence in August 2020. The marketing application is based on the outcomes from the randomized, double-blind, placebo-controlled Phase III 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 II and Phase III extension studies, and extensive natural history data. If approved, vosoritide would be the first medicine for the treatment of achondroplasia in Europe. The company remains on track to submit a New Drug Application (NDA) to the FDA in the third quarter of 2020. Vosoritide has Orphan Drug designation from the FDA and the EMA.