BioMarin Pharmaceutical Inc. recently announced that the European Medicines Agency (EMA) validated the Company’s Marketing Authorization Application (MAA) 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. The MAA review commences on August 13, 2020 ( full press here).

The company remains on track to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in the third quarter of 2020. Vosoritide has Orphan Drug designation from the FDA and the EMA.

“The extensive research conducted in the Phase 2 and 3 clinical trials, along with a natural history study, has provided more scientific and medical knowledge around skeletal dysplasia, especially achondroplasia, than we’ve ever had before,” said Melita Irving, Clinical Geneticist at Guy’s and St Thomas’ NHS Foundation Trust, London, UK and investigator for the Vosoritide clinical program at the Evelina London Children’s Hospital. “These clinical studies provide a much better understanding of how to treat the underlying conditions associated with achondroplasia more effectively and less invasively, and with no drug therapy options previously available, Vosoritide has the potential to make a meaningful impact on the daily lives of these children.”

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