FDA accepts BioMarin application for full approval of VOXZOGO

BioMarin’s VOXZOGO cleared a key regulatory hurdle when the U.S. Food and Drug Administration accepted the company’s supplemental New Drug Application for full approval in children with achondroplasia, setting a Prescription Drug User Fee Act action date of Feb. 28, 2027. The drug, also known as vosoritide, is already sold in the United States under accelerated approval, but the new filing seeks to move it into the agency’s standard approval pathway.

That distinction matters for children and families living with achondroplasia, a rare genetic skeletal disorder that limits bone growth. Accelerated approval can bring medicines to market earlier, often on the basis of surrogate measures, while full approval requires a stronger record of long-term benefit. BioMarin is asking regulators to look beyond early growth data and consider a broader body of evidence on how the treatment affects children over time.

The application is backed by long-term safety and efficacy information from three ongoing studies, including data on adult height and other outcomes that reach beyond simple linear growth. Those measures include body proportionality and arm span, endpoints that speak to how a child grows, not just how tall that child becomes. For families, that broader review could matter as much as the height numbers themselves, because achondroplasia affects the shape and balance of the skeleton as well as stature.

If the FDA grants full approval in 2027, the change would likely strengthen physician confidence and improve the drug’s standing with insurers, which often look more favorably on therapies that have completed the full approval process. It would also give families clearer expectations that VOXZOGO is not just an early-access therapy, but a treatment with a deeper evidence base supporting longer-term use.

The review also carries commercial weight for BioMarin. VOXZOGO is one of the company’s signature products and a key revenue driver in its rare-disease portfolio, and the filing comes as competition in the space has intensified. For BioMarin, the FDA’s acceptance does not settle the question of full approval, but it does establish a firm timetable for the next major regulatory decision and keeps the drug’s future on a defined path.