Rare disease biotechnology company BioMarin Pharmaceutical Inc (Nasdaq:BMRN) announced on Monday 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 28 February 2027.

The sNDA submission was supported by substantial long-term safety and efficacy data from three ongoing studies, with clinically meaningful results in growth and improvements across key skeletal growth-related measures, including proportionality and arm span. According to BioMarin, 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 fulfil the post-marketing requirement to confirm that benefit and convert to full approval, supported by long-term data.