BioMarin reports data from studies of Voxzogo

BioMarin (BMRN) Pharmaceutical announced new data from studies of VOXZOGO, or vosoritide, demonstrating meaningful impact on tibial bowing in children with achondroplasia and investigational early efficacy results in other skeletal conditions. The data were presented at the 2025 Joint Congress of the European Society for Paediatric Endocrinology, or ESPE, and the European Society of Endocrinology, or ESE, in Copenhagen, Denmark and will be shared at the Pediatric Endocrine Society Annual Meeting, or PES, in National Harbor, Maryland. An analysis of data presented at PES from the VOXZOGO Phase 2 CANOPY clinical studies in younger children assessed the impact of treatment on tibial bowing, an orthopedic complication and significant cause of pain in children with achondroplasia. Children who received VOXZOGO had a significant reduction in the magnitude of tibial bowing compared to children who received placebo. Furthermore, the researchers found that this improvement was sustained in children who received treatment for several years. The latest results from the VOXZOGO Phase 3 clinical trial reporting the longest follow-up to date demonstrated that continuous and early treatment was associated with sustained increases in annualized growth velocity.