FDA accepts sNDA to expand use of Voxzogo for injection to treat children with achondroplasia under the age of 5.- BioMarin
-BioMarin Pharmaceutical Inc., a global biotechnology company dedicated to transforming lives through genetic discovery, announced that the FDA has accepted the company's supplemental New Drug Application (sNDA) for Voxzogo (vosoritide) for injection to expand treatment in the United States to include children with achondroplasia under the age of 5.
Achondroplasia is the most common form of disproportionate short stature. The FDA has set a PDUFA target action date of October 21, 2023 for the sNDA.
The sNDA is supported by results from a Phase II randomized, double-blind, placebo-controlled clinical trial, which demonstrated similar safety and efficacy profiles in children under 5 years of age as compared to those ages 5 years and older.
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