U.S. Food and Drug Administration Approves BioMarin's VOXZOGO® (vosoritide) for Children Under 5 Years with Achondroplasia
Retrieved on:
Friday, October 20, 2023
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SAN 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.
Key Points:
- "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.
- The overall safety profile of VOXZOGO in children under 5 years of age was similar to that seen in older children.
- While similar to achondroplasia, people with hypochondroplasia typically present with milder disproportionality and less severe short stature compared to achondroplasia.