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BioMarin Plans Regulatory Submissions for Marketing Authorization of Vosoritide to Treat Children with Achondroplasia in 3Q 2020 in both US and Europe
If approved, vosoritide would be the first medicine for the treatment of Achondroplasia in the US and EU SAN RAFAEL, Calif., April 6, 2020 /PRNewswire/ --
About this update from Biomarin Pharmaceutical Inc.
[{"type":"text","content":"If approved, vosoritide would be the first medicine for the treatment of Achondroplasia in the US and EU\n\n\nSAN RAFAEL, Calif., April 6, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that based on recent meetings with health authorities in the US and Europe, the Company plans to submit marketing applications to the US Food and Drug Administration (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. \n\n \n \n \n \n \n \n\n \nThe marketing applications are based on the outcomes from the randomized, double-blind, placebo-controlled Phase 3 study evaluating the efficacy and safety of vosoritide, announced in Dec 2019, and further supported by the long-term safety and efficacy from the Phase 2 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.\n\"We have worked with the regulatory authorities throughout the design and development of our clinical program and look forward to the ongoing interactions in the evaluation of the safety and efficacy of vosoritide in children with achondroplasia,\" said Hank Fuchs, M.D., President Worldwide Research and Development at BioMarin. \"We believe that we have a strong data package that combines the gold standard of a randomized, double-blind, placebo-controlled Phase 3 study with the long-term results in the Phase 2 open label study and extensive contemporaneous natural history data to evaluate durability. We are grateful to the children and families who have participated in these studies and are contributing to the greater body of scientific data on a potential treatment for achondroplasia.\"\n\"Vosoritide is the first potential pharmacological treatment for the underlying cause of achondroplasia. It could be a medical breakthrough in providing physicians with a new tool to treat individuals with achondroplasia,\" said John A. Phillips, III, M.D., Vanderbilt University Medical Center (David T Karzon Professor of Pediatrics) and investigator for the vosoritide clinical program. \"To have such a possible treatment for...