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IndexedBuilt automatically from public databases on 2026-09-30. Not yet hand-reviewed by PLOBT.How this works →

Vosoritide

FDA approved · Rx

C-type natriuretic peptide analog

Also known as: Voxzogo, Voxzogo 0.4mg, Voxzogo 0.56mg, Voxzogo 1.2mg, BMN-111, BMN 111, vosoritida, vosoritidum, VOXZOGO 0.4mg

Evidence score
89/ 100
Strong evidence

Strong, human-grade evidence

  • Regulatory standing. FDA-approved prescription medicine
  • Human trials. Backed by controlled human studies
  • Registered human trials. 17 registered studies on ClinicalTrials.gov, most advanced Phase 3
  • 2 cited sources. Includes primary / regulatory sources

Computed with the same four public criteria as every profile. The human-evidence grade on an indexed profile is set automatically and may change when it is hand-reviewed. It is not a safety score, not a purity rating, and not medical advice, and not a claim that this compound treats, cures, or prevents any condition.

What it is

Vosoritide, sold as Voxzogo, is a modified form of C-type natriuretic peptide (CNP), a signaling molecule the body makes naturally. It binds the natriuretic peptide receptor B on growth plate cells, which is thought to counteract the overactive FGFR3 signaling that slows bone growth in achondroplasia.

What the evidence shows

Automatic summaryHuman trials

Vosoritide is FDA approved for increasing linear growth in children with achondroplasia whose growth plates are still open, supported by Phase 3 trials among 17 registered human studies. Its long-term effects on final adult height, proportions, and complications of achondroplasia are still being studied, and it is not established for other uses.

Key facts

Molecular formula
C176H290N56O51S3
Molecular weight
4103 g/mol
FDA approval
Confirmed on openFDA

Registered human trials

Live from ClinicalTrials.gov, refreshed daily.

17registered studies
Phase 3most advanced phase
4completed (sample)
3recruiting (sample)

A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months

Phase 2RecruitingNCT07126262

An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia

Phase 3Active Not RecruitingNCT03424018

A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature

Phase 2RecruitingNCT06382155

A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia

Phase 2Active Not RecruitingNCT04554940

Published literature

Live from Europe PMC. This is how much has been published, not how much is proven in humans.

134papers referencing Vosoritide

C-Type Natriuretic Peptide Analogue Therapy in Children with Achondroplasia.

N Engl J Med2019

Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial.

Lancet2020

Safe and persistent growth-promoting effects of vosoritide in children with achondroplasia: 2-year results from an open-label, phase 3 extension study.

Genet Med2021

Vosoritide therapy in children with achondroplasia aged 3-59 months: a multinational, randomised, double-blind, placebo-controlled, phase 2 trial.

Lancet Child Adolesc Health2024

Sources

Risks, side effects and study doses are only published on hand-reviewed profiles. Want this one reviewed, or spotted something wrong? Tell us.