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BioMarin Submits New Drug Application to U.S. Food and Drug Administration for Vosoritide to Treat Children with Achondroplasia
Potential first treatment for achondroplasia in the United States European Regulatory Review Began on Aug. 13 SAN RAFAEL, Calif., Aug. 20, 2020 /PRNewswire/
About this update from Biomarin Pharmaceutical Inc.
[{"type":"text","content":"Potential first treatment for achondroplasia in the United States\n European Regulatory Review Began on Aug. 13\n\n\nSAN RAFAEL, Calif., Aug. 20, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) 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. BioMarin recently announced that the European Medicines Agency (EMA) validated the Company's Marketing Authorization Application (MAA) for vosoritide on Aug. 13, 2020.\n\n \n \n \n \n \n \n\n \n\"We are grateful to the children and families who have participated in the clinical studies to evaluate the potentially first pharmacological treatment option for children with achondroplasia. We are proud of our team and clinical partners, who have conducted extensive scientific and clinical research to address the underlying cause of the condition,\" said Hank Fuchs, M.D., President, Global Research and Development at BioMarin. \"This is an important step to bring a treatment where none have existed before. We also recognize a broad range of views about treatment options, and we look forward to providing children with achondroplasia and their families a treatment choice with this potential new therapy.\"\n\"This regulatory submission is an important scientific and medical milestone for children with achondroplasia and their families,\" said Julie Hoover-Fong, MD, PhD, FACMG, Professor, McKusick-Nathans Department of Genetic Medicine and Director, Greenberg Center for Skeletal Dysplasias at Johns Hopkins University and an investigator in the vosoritide clinical program. \"The extensive clinical program for vosoritide includes important natural history information, which has contributed to the body of scientific knowledge about achondroplasia and a potential treatment option for patients.\"\n\"This regulatory submission brings our community closer to accessing the first therapeutic choice for children and families,\" said Chandler Crews, Founder of The Chandler Project. \"A well-researched drug treatment choice has the potential to be an important resource for the community and to increase our understanding of the scientif...