Voxzogo sNDA accepted for full approval in children with achondroplasia
- Sponsor
- BioMarin Pharmaceutical $BMRN
- Drug
- Voxzogo · vosoritide
- Indication
- Achondroplasia in children (label conversion from accelerated to full approval)
FDA accepted BioMarin's supplemental NDA for VOXZOGO (vosoritide) seeking full approval — converting from the 2021 accelerated approval — in children with achondroplasia. PDUFA target action date is February 28, 2027.
What this means
Voxzogo is BioMarin's once-daily injection for children with achondroplasia, the most common form of skeletal dysplasia, in which growth-plate cartilage fails to convert to bone normally. The drug is a synthetic version of a natural signaling molecule (C-type natriuretic peptide, or CNP) that pushes back against the overactive FGFR3 mutation driving achondroplasia, and has been on the market since November 2021 under accelerated approval based on annualized growth velocity data. FDA has accepted BioMarin's sNDA converting the label from accelerated to full approval, drawing on 3 long-term studies including adult-height outcomes — this would be the first achondroplasia therapy to make that conversion. A decision is expected by February 28, 2027.
From the source
SAN RAFAEL, Calif., July 13, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the U.S. 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 Feb. 28, 2027.
"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," said Greg Friberg, M.D., Executive Vice President and Chief Research & Development Officer at BioMarin. "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 three 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.
Reproduced from PRNewswire — BioMarin · view original · public domain or open-licensed government work
Drug Watch — recent news on Voxzogo
- FDA accepts sNDA for vosoritide in children with achondroplasia - Contemporary Pediatrics
- U.S. Food and Drug Administration Accepts BioMarin's Supplemental New Drug Application for Full Approval of VOXZOGO® (vosoritide) for Children With Achondroplasia - PR Newswire
- Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial - The Lancet
- Early initiation of therapy with vosoritide: case report. Description of the first Italian patient affected by achondroplasia treated with vosoritide before 2 years of age - Frontiers
- BioMarin Announces New Three-Year VOXZOGO® (vosoritide) Data in Hypochondroplasia and BMN 333 Early Results at ENDO 2026 - PR Newswire