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BioMarin Presents New Data for VOXZOGO® (vosoritide) in Children with Achondroplasia and Other Skeletal Conditions at Two International Endocrinology Meetings
New data in children under 5 years with achondroplasia showed treatment with VOXZOGO was associated with improvement in tibial bowing, a common cause of pain
About this update from Biomarin Pharmaceutical Inc.
[{"type":"text","content":"New data in children under 5 years with achondroplasia showed treatment with VOXZOGO was associated with improvement in tibial bowing, a common cause of pain and impaired function \nData from the largest retrospective, multi-year observational study of more than 600 children and adults with hypochondroplasia found significantly higher rates of comorbidities, surgeries and overall doctor visits \nSAN RAFAEL, Calif., May 12, 2025 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced new data from studies of VOXZOGO® (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 (ESPE) and the European Society of Endocrinology (ESE) in Copenhagen, Denmark and will be shared at the Pediatric Endocrine Society Annual Meeting (PES) in National Harbor, Maryland.\n\n \n \n \n \n \n \n\n \nVOXZOGO Demonstrated Positive Impact on Tibial BowingAn 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.\n\"Outcomes beyond height have long been prioritized by the dwarfism community,\" said Michael Hughes, Chair, Little People of America's (LPA) Biotech Industry Liaison Committee. \"Tibial bowing can be a source of pain and surgical intervention for some children with achondroplasia, and this research reflects an encouraging shift toward addressing a meaningful outcome to the community.\"\n\"In children with achondroplasia, tibial bowing can be painful and often requires surgical intervention,\" said Klane White, M.D., pediatric orthopedic surgeon and Rose Brown Endowed Chair of Pediatric Orthopedics at Children's Hospital Colorado. \"Through my clinical practice, I have observed meaningful improvements that early treatment initiation with VOXZOGO has offered children with acho...