BioMarin announces new research from studies of Voxzogo

BioMarin (BMRN) Pharmaceutical announced new research from studies of Voxzogo in children with achondroplasia demonstrating positive impact on important health indicators, including arm span and bone density. The data will be presented, along with new data from studies of Voxzogo in hypochondroplasia, at the Pediatric Endocrine Society’s 2026 Annual Meeting, PES, in San Francisco. Data from three ongoing long-term extension clinical trials demonstrated the impact of long-term treatment with Voxzogo on measures beyond height, including arm span and bone health. Researchers showed that arm span Z-scores improved from baseline in all age groups, and the arm span-to-height ratio also remained stable over time, showing treatment resulted in proportional skeletal growth. Children who initiated treatment with Voxzogo after age 5 also achieved a mean difference in standing height of 10.60 cm after six years of treatment and 13.59 cm after eight years of treatment, compared with untreated natural history cohorts.