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BioMarin Presents New Data on the Positive Impact of Early Treatment With VOXZOGO® (vosoritide) on Proportionality and Arm Span in Children with Achondroplasia at the 2026 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting

BMRN
  • BioMarin announced new data at the 2026 ACMG Annual Clinical Genetics Meeting showing early VOXZOGO treatment leads to durable improvements in skeletal growth, proportionality, arm span, and BMI.
  • Treatment initiated before age 2 resulted in significant, multi-year improvements in upper-to-lower body segment ratio, with differences from untreated children increasing from year one through year four.
  • Children starting VOXZOGO before age 2 gained an average of 4.7 cm in height and 0.8 Z-score improvement over four years compared to untreated peers.
  • Real-world evidence from Japan, Europe, and the U.S. confirms robust adherence and sustained growth benefits, including mean height gains of 9.91 cm at 12 months in Japanese patients and 16.7 cm at 36 months in French patients.
  • Over 5,000 infants and children across more than 50 countries have received VOXZOGO, building on more than 10,000 patient-years of data.
  • VOXZOGO remains the only approved treatment for children with achondroplasia starting at birth, with consensus guidelines recommending initiation as early as possible to maximize clinical benefit.
  • Key poster presentations at the meeting will cover long-term bone mineral density improvements, real-world healthcare utilization, and interim results from the Acorn Study regarding long-term safety.
  • VOXZOGO is a C-type natriuretic peptide analog that acts as a positive regulator of the FGFR3 signaling pathway to promote endochondral bone growth in children with open epiphyses.
  • The drug is approved in the U.S., Japan, and Australia for increasing linear growth, with EU approval for children aged 4 months and older whose epiphyses are not closed.
  • Common side effects include injection site reactions, high alkaline phosphatase levels, vomiting, joint pain, decreased blood pressure, and stomachache.
  • Serious risks include temporary blood pressure decreases, which can be mitigated by having patients eat a meal and drink fluid before administration.
  • BioMarin offers patient support through Clinical Coordinators, a caregiver mentorship program, and a U.S. doctor directory to assist families navigating achondroplasia care.