BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) presented new clinical data on its drug VOXZOGO at the Pediatric Endocrine Society's 2026 Annual Meeting in San Francisco. The research demonstrated that long-term treatment with VOXZOGO improved arm span measurements and maintained bone health in children with achondroplasia, according to the company's press release statement.

Data from three ongoing long-term extension clinical trials showed that arm span Z-scores improved from baseline across all age groups. Children who began treatment after age 5 achieved a mean difference in standing height of 10.60 cm after six years of treatment and 13.59 cm after eight years compared with untreated natural history cohorts.

A separate study of 119 children receiving VOXZOGO measured bone mineral content and bone mineral density over six years. Results showed bone mineral content increased over time while bone mineral density Z‑scores remained consistent, indicating bone health was maintained during long-term treatment.

The company also presented data from studies of VOXZOGO in hypochondroplasia. A Phase 2 study conducted by Children's National Hospital showed statistically significant improvements in bone mineral density and content after 12 months of treatment.

BioMarin plans to release topline results from its Phase 3 clinical trial of VOXZOGO in children with hypochondroplasia in the first half of 2026. The company stated it intends to submit data to health authorities in the second half of 2026 if results are positive.

VOXZOGO is currently approved to increase linear growth in children with achondroplasia in the United States, Japan, Australia and the European Union. The drug has been used by more than 5,000 children in over 50 countries since its first approval in 2021.