BioMarin Seeks Full FDA Approval for Achondroplasia Therapy VOXZOGO

The supplemental application relies on comprehensive safety and efficacy findings gathered from three ongoing clinical studies. Unlike the initial 2021 accelerated approval, which focused primarily on annualized growth velocity, this new submission presents evidence concerning broader skeletal health markers. These include improvements in body proportionality, arm span, and adult height outcomes observed over extended follow-up periods.

Greg Friberg, Executive Vice President and Chief Research and Development Officer at BioMarin, stated that the data set represents the largest body of evidence compiled for an achondroplasia medicine to date. If granted, the transition to full approval would validate the clinical benefits of the therapy as a long-term intervention for the condition, which affects roughly one in 25,000 live births. The drug, a C-type natriuretic peptide analog, is currently administered to children whose growth plates remain open.