FDA-approved

Bones & muscles

Vosoritide

EvidenceFDA approved
ClassCNP analog

Vosoritide (Voxzogo) is an FDA-approved peptide that increases growth in children with achondroplasia. Learn how it works, its side effects, and the evidence.

Reviewed by
William Maish, MD MBA MPH, Clinical Product Lead
Published
October 5, 2026
Last updated
October 5, 2026

Key takeaway

Vosoritide (sold as Voxzogo) is a modified form of C-type natriuretic peptide used to increase growth in children with achondroplasia, a genetic condition in which overactive FGFR3 signaling slows bone growth. It counteracts the overactive growth signal that restrains bone growth in the condition. It has been FDA-approved in the U.S. since 2021 and is given as a daily subcutaneous injection.

At a glance

What it isA modified analog of C-type natriuretic peptide (CNP)
Approved forAchondroplasia in children with open growth plates
Key factIn a phase 3 trial it increased annual growth velocity by about 1.6 cm per year versus placebo
RouteSubcutaneous injection, once daily
Evidence strengthFDA approved

What is vosoritide?

Vosoritide, sold under the brand Voxzogo, is a modified analog of C-type natriuretic peptide (CNP), a signaling molecule involved in bone growth. It is used to increase height in children with achondroplasia, a genetic condition caused by an overactive FGFR3 gene that slows the growth of bones at the growth plate. It was developed under the code BMN-111 and approved in the United States in 2021.

How does vosoritide work?

In achondroplasia, a mutation leaves the FGFR3 receptor constantly switched on, and its downstream signaling suppresses the growth of cartilage cells at the growth plate, limiting bone lengthening. Vosoritide binds a different receptor, natriuretic peptide receptor-B, which raises cyclic GMP inside those cartilage cells and inhibits the overactive FGFR3 pathway. By counteracting that brake, it allows more normal growth of the long bones.

Natural CNP is broken down within minutes; vosoritide is engineered to last long enough for once-daily dosing. In the phase 3 trial, children on vosoritide grew about 1.6 cm per year faster1 than those on placebo, and it is used while the growth plates remain open. A known effect is a transient drop in blood pressure, so children are given food and fluid before dosing.

Is it FDA-approved?

Yes. Vosoritide (Voxzogo) was approved2 by the FDA in 2021 for achondroplasia in children with open epiphyses (growth plates). It holds accelerated approval, based on faster annual growth, and continued approval may depend on confirmatory trials. The approved age range was initially 5 years and older, and since October 2023 it covers children of all ages with open growth plates.

What is vosoritide prescribed for?

Vosoritide is prescribed to increase linear growth in children with achondroplasia who still have open growth plates. Treatment stops once the growth plates close, since the drug works through active bone growth.

What are the side effects?

According to the prescribing information, common side effects include:

  • Injection-site reactions
  • Vomiting
  • Transient drops in blood pressure
  • Joint pain

In children under 5, rash is also common.

More serious considerations include:

  • Low blood pressure, which is managed by ensuring the child has eaten and had fluids before the dose

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Sources: FDA prescribing information for Voxzogo; the phase 3 vosoritide trial (Savarirayan et al., The Lancet, 2020, DOI: 10.1016/S0140-6736(20)31541-51), indexed on PubMed. This document is educational and does not constitute medical advice.