BioMarin plans regulatory submissions in Q3 2020 at FDA and EMA for vosoritide to treat children with achondroplasia.

BioMarin Pharmaceutical Inc. announced that based on recent meetings with health authorities in the US and Europe, the Company plans to submit marketing applications to the FDA and the European Medicines Agency (EMA) in the third quarter of 2020 for vosoritide. Vosoritide is an investigational, once daily injection analog of C-type Natriuretic Peptide (CNP) for achondroplasia, the most common form of disproportionate short stature in humans.

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The marketing applications are 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 Phase II study, ongoing extension studies, and extensive natural history data. If approved, vosoritide would be the first medicine for the treatment of Achondroplasia in the US and Europe.