BioMarin presents new data for Voxzogo (vosoritide) in children with achondroplasia and other skeletal conditions at two international endocrinology meetings

BioMarin Pharmaceutical Inc. 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.

Voxzogo Demonstrated Positive Impact on Tibial Bowing
An analysis of data presented at PES from the Voxzogo Phase II 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.

“Outcomes beyond height have long been prioritized by the dwarfism community,” said Michael Hughes, Chairman, 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.”

“In children with achondroplasia, tibial bowing can be painful and often requires surgical intervention,” said Dr. Klane White, 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 achondroplasia, including in proportionality, quality of life and now tibial bowing, which have the potential to improve physical well-being for these children into adulthood.”

The latest results from the Voxzogo Phase III clinical trial reporting the longest follow-up to date demonstrated that continuous and early treatment was associated with sustained increases in annualized growth velocity (AGV). For the first time, it was possible to model potential final height gain for children with achondroplasia if they were treated early and continuously from 6 months until final adult height (FAH). Modeling indicated that Voxzogo treatment could result in a height increase of 21.7 cm (95% Cl, 18.7-24.6) in girls and 26.4 cm (95% Cl, 22.9-29.8) in boys compared to untreated children. These data were presented at ESPE as an oral presentation and add to the totality of evidence demonstrating that early and continuous treatment with Voxzogo maximizes clinical benefits for children with achondroplasia.

See citation- Hoover-Fong J , Irving M et al. ,Long-Term Height Gain and Maintenance of Treatment Effect in Children with Achondroplasia Receiving Vosoritide. Endocrine Abstracts 2025, 110 OC7.4 DOI: 10.1530/endoabs.110.OC7.4