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BioMarin to Present Data Showing Long-Term Benefit of VOXZOGO® (vosoritide) on Growth in Children with Achondroplasia at 2023 European Society for Paediatric Endocrinology (ESPE) Meeting
Label Expansion Decisions Expected from U.S. and European Regulatory Authorities in Q4 SAN RAFAEL, Calif., Sept. 21, 2023 /PRNewswire/ -- BioMarin
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[{"type":"text","content":"Label Expansion Decisions Expected from U.S. and European Regulatory Authorities in Q4\nSAN RAFAEL, Calif., Sept. 21, 2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, today announced that new data on VOXZOGO® (vosoritide) in children with achondroplasia will be presented at the 61st Annual European Society for Paediatric Endocrinology (ESPE) Meeting in The Hague, Netherlands.\n\n \n \n \n \n \n \n\n \nIn an open-label, long-term Phase 2 extension study, VOXZOGO demonstrated consistent and durable growth benefits in young children with achondroplasia when initiated before the age of 5. Over a four-year period, children aged 2 years and above who received VOXZOGO exhibited a mean (average) height Z-score improvement exceeding 1 standard deviation compared to control groups and a height gain of more than 6 centimeters (cm) during this time period. Additionally, children under the age of 2 years, treated with VOXZOGO for three years, demonstrated substantial height gains, reflected in a mean height Z-score improvement of 0.79 standard deviations compared to controls and a height gain of more than 3 cm during this time period. Both groups of treated children demonstrated substantial restoration of height when compared to untreated children with achondroplasia.\nVOXZOGO is currently approved in Europe in children with achondroplasia who are 2 years of age and older with open growth plates. Last week, the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) 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 (growth plates) are not closed. VOXZOGO is also currently approved in the United States in children with achondroplasia who are 5 years of age and older with open growth plates. The U.S. Food and Drug Administration (FDA) has 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 U.S. to include children with achondroplasia under 5 years of age.\n\"The data presented add to the growing body of evidence demonstrating the positive effect on growth in younger childre...