The US FDA accepted BioMarin Pharmaceutical Inc.'s (Nasdaq: BMRN) supplemental New Drug Application seeking traditional approval for Voxzogo (vosoritide) in children with achondroplasia, setting a PDUFA target action date of February 28, 2027.
Voxzogo received accelerated approval in 2021 based on improvements in annualized growth velocity, a surrogate measure considered reasonably likely to predict clinical benefit. The sNDA is intended to fulfill the associated postmarketing requirement by confirming that treatment produces durable clinical benefits.
BioMarin said approval would make Voxzogo the first achondroplasia therapy to convert from accelerated to traditional approval based on a clinical package that includes adult-height outcomes. The submission draws on long-term safety and efficacy data from three ongoing studies—111-205, 111-208 and 111-302—and includes assessments of growth, body proportionality and arm span.
The company did not disclose quantitative adult-height or proportionality results with the filing acceptance. It characterized the findings as clinically meaningful and said the application contained the longest efficacy and safety follow-up submitted for an achondroplasia medicine.
Voxzogo is a C-type natriuretic peptide analog designed to counteract excessive FGFR3 signaling, the molecular mechanism that inhibits bone growth in achondroplasia. The treatment is administered by daily subcutaneous injection and is indicated in the US for pediatric patients with open growth plates.
The regulatory review comes as BioMarin faces new competition from Ascendis Pharma's Yuviwel (navepegritide), which received FDA accelerated approval on February 27, 2026, for children aged two years and older with achondroplasia and open growth plates. Yuviwel is administered once weekly, compared with once-daily dosing for Voxzogo.