BioMarin Pharmaceutical Announces New Data From Studies Of VOXZOGO In Children With Achondroplasia In Ongoing Clinical Trials And Real-World Studies

New results demonstrate early VOXZOGO treatment leads to durable and sustained improvements in skeletal growth-related health outcomes, including proportionality, arm span and body mass index, building on over 10,000 patient-years of data  

VOXZOGO is the only approved treatment for children with achondroplasia starting at birth and has the most extensive published clinical evidence, including in measures beyond growth, among all achondroplasia treatments

SAN RAFAEL, Calif., March 12, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced positive new data from studies of VOXZOGO® (vosoritide) in children with achondroplasia in ongoing clinical trials and real-world studies. The latest findings reinforce the importance of early treatment and the well-known, established efficacy and safety of VOXZOGO. The data will be presented at the 2026 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting in Baltimore.

Early Treatment, Maximum Impact in Children Who Start Treatment Before Age 2

New data will be presented from a study of children who started VOXZOGO before age 2, which showed that treatment was associated with durable, multi-year growth improvements, including body proportionality and arm span compared to untreated children, as well as improvements in height over time.

For proportionality, measured by change from baseline in upper‑to‑lower body segment ratio (ULBR), the least squares mean difference versus untreated children showed improvement by year one in children initiating VOXZOGO under age 2, and this difference continued to improve each year through year four. Specifically, the least squares mean difference versus untreated children in year one was −0.33 (p=0.0005) in children initiating VOXZOGO under age 2, and −0.10 (p=0.042) in those initiating treatment with VOXZOGO under 6 months; by year four, this trend continued, with a least squares mean difference of −0.53 (p<0.0001) in children initiating VOXZOGO under age 2 compared to untreated children, and −0.23 (p=0.0003) in those who initiated treatment with VOXZOGO under 6 months.

Additional analyses further emphasize that these children showed durable improvements in height while maintaining stable arm‑to‑height ratios, consistent with arm span improvements compared to untreated children, which is particularly relevant in early life. Researchers also saw significant improvements in body mass index (BMI) in children who initiated treatment when compared to untreated children.