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European Medicines Agency Validates Application for Extension of Indication for VOXZOGO® (vosoritide) for injection to Treat Children with Achondroplasia Under the Age of 2
BioMarin Submits Supplemental New Drug Application to U.S. Food and Drug Administration to Expand Label to Treat Children with Achondroplasia Under the Age of
About this update from Biomarin Pharmaceutical Inc.
[{"type":"text","content":"BioMarin Submits Supplemental New Drug Application to U.S. Food and Drug Administration to Expand Label to Treat Children with Achondroplasia Under the Age of 5\nSubmissions Based on Favorable Results from Global Randomized Phase 2 Study in Infants and Young Children Demonstrating Increase of Growth Velocity in All Cohorts by Age\nPotential to Benefit More than 1,000 Eligible Children in U.S. and Europe\nAction by Health Authorities Expected in 2H 2023\nSAN RAFAEL, Calif., Jan. 3, 2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, announced today that the European Medicines Agency (EMA) validated its Type II Variation application to extend the indication for VOXZOGO® (vosoritide) for injection to treat children with achondroplasia under the age of 2. Validation confirms the submission is complete and begins the EMA's review process. The Company also announced that it submitted a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) to treat children with achondroplasia under the age of 5. Approval of the submissions would mean VOXZOGO could potentially be prescribed as early as birth with more than 1,000 additional children eligible for treatment for achondroplasia, the most common form of disproportionate short stature in humans. The Company anticipates action by health authorities in the second half of 2023.\n\n \n \n \n \n \n \n\n \nBioMarin Submits Supplemental Applications for VOXZOGO Indication Extensions for Younger Children; EU ValidatesThe supplemental marketing applications are based on the outcomes from a Phase 2 randomized, double-blind, placebo-controlled clinical trial to evaluate the safety and efficacy of VOXZOGO™ (vosoritide) for injection in infants and children aged 3 months to less than five years old.\n\"We are pleased by results showing an increase in growth velocity across all age cohorts. These data are very encouraging and supportive of expanding the indication to younger children,\" said Hank Fuchs, M.D., President, Worldwide Research and Development at BioMarin. \"We look forward to further dialogue with health authorities in the U.S. and Europe to review these submissions to expand access to VOXZOGO treatment for this younger age group. Our hope is that children ar...