FDA Reviews Application for Full Approval of Achondroplasia Treatment

The U.S. FDA has accepted BioMarin's supplemental New Drug Application for VOXZOGO, a treatment for children with achondroplasia. The agency is targeting a decision date of February 28, 2027, based on long-term safety and efficacy data from ongoing studies. This move could lead to full approval for the therapy, offering a significant development for patients.