BioMarin Pharmaceutical recently announced that the US Food and Drug Administration (FDA) has accepted the company's supplemental New Drug Application (sNDA) for VOXZOGO (vosoritide) for full approval in children with achondroplasia. The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of February 28, 2027.

Greg Friberg, MD, Executive Vice President (EVP) and Chief Research & Development Officer, BioMarin, said, "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 annualised growth velocity in children with this condition. If approved, VOXZOGO would be the first therapy for achondroplasia to convert from accelerated approval to traditional approval based on a comprehensive clinical data package, including adult height outcomes and other clinical measures evaluated over extended follow-up."

The sNDA submission was supported by substantial long-term safety and efficacy data from 3 ongoing studies (111-205, 111-208 and 111-302), including clinically meaningful results in growth and improvements across key skeletal growth-related measures, including proportionality and arm span. The full package submitted to the FDA included the longest efficacy and safety data of any medicine studied in achondroplasia.

VOXZOGO received FDA accelerated approval in 2021, a pathway enabling faster patient access based on measures reasonably likely to predict clinical benefit. This sNDA is intended to fulfill the postmarketing requirement to confirm that benefit and convert to full approval, supported by long-term data from 3 ongoing studies demonstrating clinically meaningful improvements in growth and skeletal health outcomes in children with achondroplasia.