BioMarin received Health Canada Notice of Compliance with Conditions (NOC/c) for Voxzogo on February 11, 2026, making it the only approved medicine for eligible achondroplasia patients in Canada2
Recent positive data from Voxzogo clinical trials and real-world studies were presented at the 2026 American College of Medical Genetics and Genomics Annual Clinical Genetics Meeting in Baltimore, reinforcing the importance of early treatment and established efficacy and safety4
A Phase 2 study of Voxzogo in infants and young children (aged 0 to less than 5 years) showed serious adverse events were higher in the placebo group (18%) compared to Voxzogo-treated children (7%), with the most common adverse events being mild and self-limiting injection site reactions13
BioMarin presented new data demonstrating that early Voxzogo treatment leads to durable and sustained improvements in skeletal growth-related health outcomes, including positive impacts on proportionality and arm span in children with achondroplasia6
The search results do not contain information about BioMarin halting Phase 2 Voxzogo dosing in certain patients over safety concerns; available data indicates generally favorable safety profiles across clinical studies
Sources:
1. https://www.biomarin.com/news/press-releases/biomarin-announces-favorable-results-from-global-phase-2-study-of-voxzogo-vosoritide-for-injection-in-infants-and-young-children-with-achondroplasia-at-the-endocrine-society-annual-meeting/
2. https://www.biospace.com/press-releases/biomarin-receives-health-canada-notice-of-compliance-with-conditions-for-voxzogo-for-achondroplasia
3. https://www.biomarin.com/news/press-releases/european-medicines-agency-validates-application-for-extension-of-indication-for-voxzogo-vosoritide-for-injection-to-treat-children-with-achondroplasia-under-the-age-of-2/
4. https://intellectia.ai/news/stock/biomarin-announces-positive-data-from-voxzogo-clinical-trials
6. https://www.biomarin.com/news/press-releases/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-med/