FDA Accepts BioMarin's Supplemental Application for Full Approval of VOXZOGO for Achondroplasia

BioMarin Pharmaceutical Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) for VOXZOGO® (vosoritide) seeking full approval for the treatment of achondroplasia in children. The FDA has assigned a target action date of February 28, 2027, under the Prescription Drug User Fee Act (PDUFA).

The application is supported by long-term safety and efficacy data from three ongoing studies. These studies include data on adult height and other clinical outcomes beyond linear growth, assessed over extended follow-up periods. VOXZOGO initially received accelerated approval from the FDA in 2021, a pathway designed to provide earlier patient access to the therapy.

Achondroplasia is the most common form of skeletal dysplasia, characterized by disproportionate short stature. The condition results from a genetic mutation that negatively regulates endochondral ossification. The sNDA aims to confirm the therapy's clinical benefit based on the comprehensive long-term data collected.

BioMarin stated that this submission is backed by the largest body of evidence for any medicine in achondroplasia. If approved, VOXZOGO would represent the first therapy for achondroplasia to transition from accelerated to traditional approval, based on a robust clinical data package.