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BioMarin Receives Positive CHMP Opinion in Europe to Expand Use of VOXZOGO® (vosoritide) to Treat Children Aged 4 Months and Older with Achondroplasia
European Commission Approval Decision Expected Q4 2023 Opinion Based on Positive Results from Global Phase 2 and Ongoing Extension Study U.S. Food and Drug
About this update from Biomarin Pharmaceutical Inc.
[{"type":"text","content":"European Commission Approval Decision Expected Q4 2023\nOpinion Based on Positive Results from Global Phase 2 and Ongoing Extension Study \nU.S. Food and Drug Administration PDUFA Target Action Date for Supplemental New Drug Application for VOXZOGO for Children Under 5 is Oct. 21, 2023\nSAN RAFAEL, Calif., Sept. 15, 2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, today announced that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending marketing authorization to expand the indication for VOXZOGO® (vosoritide) for injection to treat children with achondroplasia aged 4 months and older whose epiphyses (growth plates) are not closed. The diagnosis of achondroplasia should be confirmed by appropriate genetic testing. A final approval decision, typically consistent with the CHMP recommendation, is expected from the European Commission in the fourth quarter of 2023. VOXZOGO is currently approved in Europe in children with achondroplasia who are 2 years of age and older with open growth plates.\n\n \n \n \n \n \n \n\n \nThe U.S. Food and Drug Administration (FDA) has also set a PDUFA Target Action Date of Oct. 21, 2023, for the company's Supplemental New Drug Application (sNDA) for VOXZOGO to expand treatment in the United States to include children with achondroplasia under the age of 5 years. VOXZOGO is currently approved in the U.S. in children with achondroplasia who are 5 years of age and older with open growth plates.\n\"The expansion of VOXZOGO to allow physicians to begin treatment earlier offers the possibility of increased growth and other potential important long-term benefits,\" said Hank Fuchs, M.D., president of Worldwide Research and Development at BioMarin. \"We look forward to the final approval decision from the European Commission, as well as a decision from the FDA, later this year. These approvals have the potential to further advance care by reaching very young children with achondroplasia.\"\nSince the introduction of VOXZOGO in 2021, the company has seen strong demand for the medicine worldwide. Over the last few weeks, BioMarin has increased fill-finish commitments and is continuing to work with the company's fill-finish pr...