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FDA Accepts BioMarin's Supplemental New Drug Application to Expand Use of VOXZOGO® (vosoritide) for Injection to Treat Children with Achondroplasia Under the Age of 5
The Supplemental New Drug Application is Based on Positive Results from Global Randomized Phase 2 Study in Infants and Young Children FDA set PDUFA Target
About this update from Biomarin Pharmaceutical Inc.
[{"type":"text","content":"The Supplemental New Drug Application is Based on Positive Results from Global Randomized Phase 2 Study in Infants and Young Children\nFDA set PDUFA Target Action Date of October 21, 2023\nSAN RAFAEL, Calif., March 7, 2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, announced today that the U.S. Food and Drug Administration (FDA) has accepted the company's supplemental New Drug Application (sNDA) for VOXZOGO® (vosoritide) for injection to expand treatment in the United States to include children with achondroplasia under the age of 5. Achondroplasia is the most common form of disproportionate short stature.\n\n \n \n \n \n \n \n\n \nThe FDA has set a PDUFA target action date of October 21, 2023 for the sNDA.\n\"We are pleased that the FDA has accepted our sNDA and are working closely with the agency to facilitate completion of the review in a timely manner,\" said Hank Fuchs, M.D., president, Worldwide Research and Development at BioMarin. \"There are currently no approved pharmacological treatments in the United States for children under 5 with achondroplasia and this approval could potentially extend access to all children with achondroplasia, whose growth plates are not closed.\"\nThe sNDA is supported by results from a Phase 2 randomized, double-blind, placebo-controlled clinical trial, which demonstrated similar safety and efficacy profiles in children under 5 years of age as compared to those ages 5 years and older.\nIn January, the European Medicines Agency (EMA) validated BioMarin's application for extension of indication for VOXZOGO to treat children with achondroplasia under the age of 2. Approval of the submissions would mean VOXZOGO could potentially be prescribed as early as birth for more than 1,000 additional children eligible for treatment for achondroplasia.\nVOXZOGO is the first FDA and EMA approved treatment for children with achondroplasia with open epiphyses (bone growth plates).\nAbout VOXZOGO® (vosoritide) for Injection\nIn patients with achondroplasia, endochondral bone growth, an essential process by which bone tissue is created, is negatively regulated due to a gain of function mutation in fibroblast growth factor receptor 3 gene (FGFR3). VOXZOGO, a C-type natriuretic peptide (CNP) analog, represen...