Content reviewed by clinical research staff

Last reviewed: March 2026Sources: PubMed, FDA, WADA Prohibited List

Evidence graded using the PeptideScholar A-D system

Vosoritide

AFDA Approved
70
Good Credibility
1 cited studyy · Evidence level A

C-type natriuretic peptide analog · 39 amino acids · Brand: Voxzogo

FDA ApprovedPrescription Required

Vosoritide is a recombinant analog of C-type natriuretic peptide (CNP) approved by the FDA for achondroplasia in children aged 5 years and older with open growth plates. It is the first and only disease-modifying therapy for the most common form of dwarfism.

Mechanism of Action

Achondroplasia is caused by a gain-of-function mutation in the FGFR3 gene, which suppresses endochondral bone growth. Vosoritide binds to natriuretic peptide receptor B (NPR-B), activating the GC-B/cGMP pathway that counteracts FGFR3 signaling. This promotes chondrocyte proliferation and differentiation at the growth plate, increasing longitudinal bone growth.

Benefits

  • First disease-modifying therapy for achondroplasia[1]
  • Significantly increases annual growth velocity in children[1]
  • Improves body proportions over time
  • Once-daily subcutaneous injection (home administration)
Not Medical Advice — Research-Reported Information Only

This content is for informational purposes only and does not constitute medical advice.

Vosoritide — Dosing in Published Research

Reported Routes: Subcutaneous injection
15 mcg/kg SC once daily. Must be administered at approximately the same time each day. Requires monitoring of growth velocity, body proportions, and blood pressure. Treatment stops when growth plates close (epiphyseal fusion).

The dosing information above is sourced from published research literature and clinical trials. These are not recommendations. Individual responses vary. Always consult a healthcare provider before considering any peptide-based therapy.

Side Effects

  • Injection site reactions (erythema, swelling, pain)
  • Vomiting
  • Transient decrease in blood pressure (due to natriuretic peptide vasodilation)[1]
  • Headache
  • Potential for disproportionate growth of limbs vs trunk (requires monitoring)

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Approved Treatment Routing

Vosoritide in PeptideScholar's current approved-treatment dataset

Vosoritide is currently modeled on this site as an approved treatment path for: Achondroplasia in children aged 5 years and older with open epiphyses (growth plates). Brand names in the current dataset: Voxzogo.

Validation Note
This block reflects the site's current structured treatment data and internal routing logic. It is not a substitute for checking current prescribing, labeling, payer, or local regulatory details.

Research & Evidence

RCTN Engl J Med, 2019

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

Vosoritide 15 mcg/kg daily significantly increased mean annualized growth velocity by 1.57 cm/year vs placebo in children with achondroplasia over 52 weeks

PMID: 31269546

References

  1. 1. C-Type Natriuretic Peptide Analogue Therapy in Children with Achondroplasia.. N Engl J Med, 2019. Vosoritide 15 mcg/kg daily significantly increased mean annualized growth velocity by 1.57 cm/year vs placebo in children with achondroplasia over 52 weeks [PMID: 31269546]

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Vosoritide FAQ

Medical Disclaimer

This content is for informational and educational purposes only and does not constitute medical advice, diagnosis, or treatment recommendations.

Always consult a qualified healthcare provider before starting, stopping, or modifying any treatment. Do not disregard professional medical advice based on information found on this site.

No claims of therapeutic efficacy are made for substances that are not FDA-approved for the discussed indications. Research citations reflect published findings and do not imply endorsement.

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