As of September 2026, vosoritide is approved in the US and the EU to increase growth in children with achondroplasia whose growth plates are open. The approval rested on a year-long randomized trial in 121 children. Effects on final adult height are still being followed. A vial that did not come from a pharmacy was not in that trial, and its outside tells you nothing about what it holds.
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What is vosoritide?
Vosoritide is a lab-made version of C-type natriuretic peptide (CNP), a natural hormone that helps regulate bone growth. BioMarin developed it to counter the overactive FGFR3 signal that causes achondroplasia, the most common form of dwarfism. The EMA approved it in August 2021 and the FDA in November 2021.
Does vosoritide work?
To increase growth in children with achondroplasia, the FDA and the EMA reviewed the trials and approved it. The approval covers children whose growth plates are still open.
What we found. 2 completed randomized trials counted on the record. The study list names one of them, the Phase 3 trial in 121 children. Its other row is open-label follow-up that is still running, so the second counted trial is not listed.
| Study | Tested in | Status | What it shows |
|---|---|---|---|
| Main Phase 3 trial in 121 children with achondroplasia | People | Completed. The basis for approval | Children on vosoritide grew about 1.57 cm a year faster than those on placebo, over 52 weeks |
| Long-term extension studies | People | Ongoing, open-label | Growth gains are reported to continue over multiple years of follow-up |
Is vosoritide safe?
In the trials, safety concerns included reactions where the medicine is given, vomiting and drops in blood pressure. Long-term effects are still being followed in extension studies: final adult height, joint and spine complications, and heart and blood vessel outcomes over a full childhood. The record says those data are not yet fully mature. That is the picture for Voxzogo. The vial section asks what a vial from anywhere else contains.
Is vosoritide legal?
- United States
- Approved by the FDA in November 2021 as Voxzogo, to increase growth in children with achondroplasia.
- European Union
- Approved by the EMA in August 2021 for the same use.
- Sport
- Not named on WADA's list. That is not clearance.
Not medical advice. Approval and sport status as of . Medical disclaimer
What is in a vial sold as vosoritide?
The Phase 3 trial above was the basis for approving Voxzogo. The record says vosoritide is used only in the care of children with achondroplasia, and it shows no sign of sale outside pharmacies. If a vial reached you another way, that trial did not test it, and the checklist lists what to ask the seller.
What don't we know?
- What it does to final adult height. Extension studies are still following that.
- Whether it changes joint and spine complications over a full childhood.
- What it does to heart and blood vessel outcomes over the same span.
- What is in any vial that did not come from a pharmacy.
The other side
Vosoritide comes with a prescription and specialist care for children with achondroplasia. In its main trial, children grew about 1.57 cm a year faster than those on placebo. What that adds up to by adulthood is still being followed.
What would change this record?
- Multi-year data from the extension studies and from monitoring after approval that confirm lasting height gains and long-term safety.
Three questions to ask next
- What does 'research use only' actually mean?
- How can you tell if a peptide vendor is legitimate?
- How do you read a certificate of analysis?
Ask better questions.
More questions about vosoritide
What is vosoritide used for?
- To increase growth in children with achondroplasia whose growth plates are still open. That is its approved use in the US and the EU.
Is vosoritide the same as Voxzogo?
- Voxzogo is the brand name for vosoritide.
Does vosoritide make children taller as adults?
- Nobody knows yet. The main trial ran for 52 weeks, and effects on final adult height are still being followed in extension studies.
Is vosoritide used in adults?
- Not on this record. It says vosoritide is used only in the care of children with achondroplasia, and the approval covers children with open growth plates.
Is vosoritide banned in sport?
- It is not named on WADA's list. The record gives no sport source for that. If you compete, check the current List.
Record checked on . Each source shows the date it was checked.
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