BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) has announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) for VOXZOGO® (vosoritide) aimed at achieving full approval for treating children with achondroplasia.
The application is backed by extensive long-term safety and efficacy data from three ongoing studies, which have shown significant improvements in growth and skeletal health measures.
The FDA has set a target action date of February 28, 2027, under the Prescription Drug User Fee Act.
Achondroplasia, a genetic disorder caused by a mutation in the FGFR3 gene, is the most common form of skeletal dysplasia, leading to disproportionate short stature and affecting bone growth and structure.
VOXZOGO initially received accelerated approval from the FDA in 2021, allowing for earlier patient access based on preliminary indicators of clinical benefit. The current sNDA seeks to confirm these benefits with long-term data, thereby converting the drug’s status to full approval.
Greg Friberg, M.D., Executive Vice President and Chief Research & Development Officer at BioMarin, commented, “This submission for VOXZOGO is supported by the largest body of evidence for any medicine in achondroplasia, reflecting BioMarin’s long-standing commitment to advancing the science of skeletal growth. The clinical data demonstrate meaningful improvements across multiple skeletal growth-related measures beyond annualized growth velocity in children with this condition.”
