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U.S. Food and Drug Administration Approves BioMarin's VOXZOGO® (vosoritide) for Children Under 5 Years with Achondroplasia
Expanded Indication in the U.S. Now Includes Children of all ages with Achondroplasia SAN RAFAEL, Calif., Oct. 20, 2023 /PRNewswire/ -- BioMarin
About this update from Biomarin Pharmaceutical Inc.
[{"type":"text","content":"Expanded Indication in the U.S. Now Includes Children of all ages with Achondroplasia \nSAN RAFAEL, Calif., Oct. 20, 2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, today announced that the U.S. Food and Drug Administration (FDA) has approved the supplemental New Drug Application (sNDA) for VOXZOGO® (vosoritide) to increase linear growth in pediatric patients with achondroplasia with open epiphyses (growth plates). This indication is approved under accelerated approval based on an improvement in annualized growth velocity. Previously, VOXZOGO was indicated for children who were 5 years of age and older. This expanded indication now includes children of all ages with open growth plates.\n\n \n \n \n \n \n \n\n \n\"We are pleased that VOXZOGO is now available for children of all ages with achondroplasia,\" said Hank Fuchs, M.D., president of Worldwide Research and Development at BioMarin. \"We are grateful for the collaboration of the achondroplasia community, physicians, and the children and their families who have played a crucial role in advancing this clinical program. We also look forward to further understanding the potential role of VOXZOGO in other genetic short stature conditions, including hypochondroplasia.\"\n\"VOXZOGO is the only approved treatment for children with achondroplasia. Until now, it has only been approved in the U.S. for children aged 5 and older with open growth plates,\" said William Wilcox, M.D., professor of human genetics at Emory University. \"I am delighted that VOXZOGO is now approved for younger children where we hope to have potentially greater impact by starting treatment earlier and, as a result, a much longer treatment window.\"\nBioMarin conducted a randomized, double-blind, placebo-controlled Phase 2 clinical trial evaluating the safety and efficacy of VOXZOGO in children aged 5 and under (Study 111-206). Based on the results of this trial, together with evidence from the adequate and well controlled Phase 3 study in pediatric patients aged 5 years and older (Study 111-301), safety and effectiveness of VOXZOGO have been established in pediatric patients of all ages for the improvement in linear growth in children with achondroplasia with open epiphyses. The overall safety profile of V...