BioMarin Pharmaceutical Inc. presented new data on the long-term effects of VOXZOGO (vosoritide) at the Pediatric Endocrine Society (PES) 2026 Annual Meeting. The data, based on over 10 years of clinical research, demonstrate sustained improvements in arm span, bone health, and growth in children with achondroplasia, the most common form of dwarfism.
VOXZOGO is the only approved treatment for children with achondroplasia starting from birth. The new findings highlight its potential to address key complications associated with the condition, including reduced arm span and impaired bone density. Researchers reported that children receiving continuous treatment showed significant gains in arm span compared to natural history controls.
The study also assessed bone health markers, indicating that long-term use of vosoritide may improve bone mineral density and reduce the risk of fractures. Growth velocity remained above baseline for most patients, with a favorable safety profile consistent with previous trials. BioMarin plans to submit these data for regulatory review to support expanded labeling.
These results reinforce the role of VOXZOGO as a foundational therapy for achondroplasia, offering hope for improved functional outcomes and quality of life. Further analyses are ongoing to evaluate long-term effects into adulthood.
