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BioMarin Presents New Data on the Effect of Long-Term Treatment with VOXZOGO® (vosoritide) on Arm Span, Bone Health and Growth in Children With Achondroplasia at the Pediatric Endocrine Society's 2026 Annual Meeting

BMRN
  • BioMarin announced new long-term data for VOXZOGO in children with achondroplasia demonstrating improvements in arm span, bone mineral content, & bone mineral density.
  • Data from three long-term extension trials showed arm span Z-scores improved across all age groups while the arm span-to-height ratio remained stable.
  • Children initiating treatment after age 5 achieved a mean height difference of 10.60 cm after six years & 13.59 cm after eight years compared to untreated cohorts.
  • A study of 119 children confirmed that long-term VOXZOGO treatment increases bone mineral content while maintaining consistent bone mineral density Z-scores.
  • New data for VOXZOGO in hypochondroplasia will be presented at the Pediatric Endocrine Society's 2026 Annual Meeting in San Francisco.
  • A Phase 2 study in hypochondroplasia showed statistically significant improvements in total body minus head bone mineral density & bone mineral content after 12 months.
  • Topline results from the pivotal Phase 3 CANOPY-HCH-3 trial for hypochondroplasia are expected in the first half of 2026.
  • BioMarin plans to submit regulatory data for the hypochondroplasia indication in the second half of 2026 if Phase 3 results are positive.
  • VOXZOGO remains the only approved treatment for children with achondroplasia starting at birth, with over 10 years of clinical research supporting its long-term use.
  • The drug has helped more than 5,000 infants & children in over 50 countries since its first approval in 2021.
  • VOXZOGO is approved in the U.S., Japan, Australia, & the EU for children with achondroplasia who have open growth plates.
  • Common side effects include injection site reactions, elevated alkaline phosphatase, vomiting, joint pain, decreased blood pressure, & stomachache.
  • Patients are advised to eat a meal & drink 8 to 10 ounces of fluid within one hour before dosing to reduce the risk of temporary blood pressure decreases.
  • BioMarin offers patient support services including caregiver mentorship, a doctor directory, & clinical coordinators to assist families.