BioMarin receives positive CHMP opinion in Europe to expand use of VOXZOGO

BioMarin Pharmaceutical announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use has adopted a positive opinion recommending marketing authorization to expand the indication for VOXZOGO for injection to treat children with achondroplasia aged 4 months and older whose epiphyses 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. The U.S. FDA has also set a PDUFA Target Action Date of Oct. 21, 2023, for the company’s Supplemental New Drug Application 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.