Plain-English fact sheet
Vosoritide
Also known as Voxzogo
Vosoritide is an FDA-approved targeted peptide for children with achondroplasia and open growth plates, under accelerated approval based on growth velocity.
Quick answer
Vosoritide is FDA approved as Voxzogo to increase linear growth in children with achondroplasia whose growth plates remain open. It is not approved as a height enhancer for children without achondroplasia or after growth plates close.
By the PeptideFactSheets Editorial Team. Claims are source-checked under our editorial policy; clinician review is identified only when a named reviewer is shown.
What is Vosoritide?
Vosoritide is an analog of C-type natriuretic peptide that counteracts overactive FGFR3 signaling, the central growth-plate mechanism in achondroplasia.
The approval is based on increased annualized growth velocity; final adult height and broader functional outcomes are still being studied.
Why are people interested in it?
It targets the biology of the most common form of disproportionate short stature rather than supplying growth hormone.
Its accelerated-approval endpoint makes the distinction between faster childhood growth and confirmed long-term clinical benefit important.
Current regulatory status
Voxzogo is FDA approved under accelerated approval to increase linear growth in pediatric patients with achondroplasia and open epiphyses. The FDA removed the original minimum-age restriction in October 2023.
What is it approved for?
- Increasing linear growth in pediatric patients with achondroplasia and open growth plates
What is it being studied for?
Investigational areas
- Final adult height and broader long-term clinical outcomes
- Other genetically defined skeletal dysplasias
Evidence snapshot
A randomized Phase 3 trial demonstrated improved annualized growth velocity. Evidence for final adult height and the full set of long-term health and functional outcomes is still developing.
Potential benefits being researched
- The pivotal trial found a greater increase in annualized growth velocity than placebo over one year.
- Extension studies suggest the growth-velocity effect can persist, but confirmatory final-height evidence remains important.
Potential does not mean proven. Study design, population, endpoint, and regulatory review matter.
Known or possible risks
- The label warns about temporary decreases in blood pressure.
- Common adverse reactions include injection-site reactions, rash, vomiting, joint pain, decreased blood pressure, and gastroenteritis.
- Evidence in some age groups and medical situations is smaller than in the original pivotal population.
What we still do not know
- The magnitude of any change in final adult height
- Long-term effects on function, pain, surgery, body proportions, and quality of life
- Rare harms that may emerge with longer and earlier treatment
- Whether findings extend to skeletal conditions other than achondroplasia
Plain-English takeaway
Vosoritide is a targeted rare-disease peptide with a proven effect on childhood growth velocity. The unresolved question is how that translates into final height and broader long-term outcomes.
Research and reference links
Use these primary and reputable sources to verify status and read beyond this summary. Trial registries may list studies without proving a benefit.
- 1FDA prescribing information: Voxzogo
Current indication, accelerated-approval basis, warnings, and adverse reactions.
- 2FDA: ongoing vosoritide accelerated approval
FDA record of the confirmatory final-adult-height requirement.
- 3Randomized Phase 3 vosoritide trial
Pivotal placebo-controlled trial of annualized growth velocity in children with achondroplasia.
- 4Randomized trial in children younger than 5
Controlled study informing the expanded pediatric indication.