VOXZOGO (Vosoritide)

July 13, 2026: BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) said the FDA accepted its supplemental NDA (sNDA) for VOXZOGO (vosoritide) seeking full (traditional) approval in children with achondroplasia. The FDA set a PDUFA target action date of Feb. 28, 2027.

The sNDA is intended to satisfy the postmarketing requirement tied to VOXZOGO’s 2021 FDA accelerated approval, which was based on improvement in annualized growth velocity. The submission is supported by long-term safety and efficacy data from three ongoing studies (111-205, 111-208, and 111-302), including adult height and additional outcomes beyond linear growth, such as body proportionality and arm span, assessed over extended follow-up. BioMarin stated the package includes the longest efficacy and safety data for any medicine studied in achondroplasia and is aimed at confirming clinical benefit to convert accelerated approval to full approval.