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BioMarin Submits Marketing Authorization Application to European Medicines Agency for Vosoritide to Treat Children with Achondroplasia
If approved, vosoritide would be the first medicine to treat Achondroplasia in EU SAN RAFAEL, Calif., July 23, 2020 /PRNewswire/ -- BioMarin Pharmaceutical
About this update from Biomarin Pharmaceutical Inc.
[{"type":"text","content":"If approved, vosoritide would be the first medicine to treat Achondroplasia in EU\n\n\n SAN RAFAEL, Calif., July 23, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today 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. \n\n \n \n \n \n \n \n\n \nThe marketing application is based on the outcomes from the randomized, 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.\nThe 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. \n\"Years of scientific research have led to this important point in the development of the potentially first pharmacological treatment option for children with achondroplasia. We have worked alongside patient advocacy groups from around the world throughout the development, and we appreciate the implications of developing a treatment option for this community, recognizing that this potential new treatment would offer a choice for families who have a child with achondroplasia,\" said Hank Fuchs, M.D., President Worldwide Research and Development at BioMarin. \"Our goal is to provide a treatment option that addresses the underlying cause of the condition and over time demonstrate a reduction of complications that may result from achondroplasia. We respect the depth and breadth of views among the community about treatment options and have sought to be scientifically rigorous in providing a robust data set for regulators to evaluate the safety and efficacy of vosoritide. We remain grateful to the physicians and families who have participa...