Is Vosoritide safe?
Trial and label data in children
Injection-site reactions were the most common adverse events. They include redness, swelling and hives. Most children had them at some point. In the phase 3 trial, vomiting was more frequent than on placebo. So were brief drops in blood pressure with dizziness or nausea. The label advises giving the injection with food and fluids to reduce this. According to the label, two children on vosoritide stopped treatment because of pain and anxiety with the injections. 8 of 60 had brief drops in blood pressure. On placebo it was 3 of 61. 12
The label notes that growth plate closure should be confirmed before stopping. The drug is not expected to work after the plates have fused. Extension studies are tracking long-term effects on bone quality, blood pressure and other tissues that respond to CNP. 2
More on Vosoritide
Citations
- Savarirayan 2020, Lancet: Once-daily subcutaneous vosoritide in children with achondroplasia, randomized phase 3 trial doi.org
- FDA 2024: Voxzogo (vosoritide) prescribing information accessdata.fda.gov
- Dauber 2026, NEJM Evidence: a phase 3 trial of vosoritide in children with hypochondroplasia pubmed.ncbi.nlm.nih.gov
- BioMarin 2026: phase 3 hypochondroplasia results published in NEJM Evidence, supplemental application submitted to the FDA biomarin.com
Reference information only, not medical advice. Drawn from the Vosoritide article, fact-checked against the cited sources. No dosing anywhere on this site.