Recovery ⏱ Half-life: ~25-30 minutes

Vosoritide

Vosoritide (Voxzogo, Modified CNP Analog)

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Half-Life
~25-30 minutes
Purity
Pharmaceutical grade
Mol. Weight
4,105 Da
Form
Lyophilized powder for reconstitution

What is Vosoritide?

Vosoritide (Voxzogo) is the first and only FDA-approved therapy for achondroplasia, the most common form of dwarfism. It represents a precision medicine approach — targeting the specific molecular pathway (overactive FGFR3) responsible for impaired bone growth. Its approval was a milestone for the rare disease community and for peptide therapeutics.

Dosage Information (Research Use)

15 mcg/kg SC once daily. For pediatric patients with achondroplasia and open growth plates. Prescription medication — specialist use only.

Reconstitution & Handling

Reconstitute lyophilized powder with provided diluent. Administer within 3 hours.

Half-Life & Pharmacokinetics

~25-30 minutes

Reported Observations in Literature

Injection site reactions (35%), vomiting (27%), transient blood pressure decreases (dose-dependent, usually asymptomatic), injection site erythema. Monitor blood pressure before and 30 minutes after injection during treatment initiation.

Key Research References

  • Savarirayan R et al. “Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial.” Lancet. 2020;396:684-92

How Vosoritide Works

Vosoritide is a 39-amino acid analog of C-type natriuretic peptide (CNP) with an extended half-life achieved by adding amino acid residues to the native sequence. It activates natriuretic peptide receptor B (NPR-B), which counteracts the overactive FGFR3 signaling that inhibits bone growth in achondroplasia. By antagonizing FGFR3-mediated growth plate suppression, vosoritide restores more normal endochondral ossification.

Research Applications

🔬 Achondroplasia treatment
🔬 Endochondral ossification
🔬 FGFR3 signaling modulation

Research Findings

The Phase 3 trial showed an increase of 1.57 cm/year in annualized growth velocity compared to placebo in children aged 5-18 years. The effect was sustained over 5+ years of open-label extension. FDA-approved in 2021 as the first drug for achondroplasia.

Dosage & Administration

15 mcg/kg SC once daily. For pediatric patients with achondroplasia and open growth plates. Prescription medication — specialist use only.

Safety & Side Effects

Injection site reactions (35%), vomiting (27%), transient blood pressure decreases (dose-dependent, usually asymptomatic), injection site erythema. Monitor blood pressure before and 30 minutes after injection during treatment initiation.

Important: All safety information is derived from published research, primarily animal studies. No controlled human clinical trial data exists unless explicitly noted. This compound is sold for research purposes only.

Quick Facts

Molecular Weight 4,105 Da
Half-Life ~25-30 minutes
Purity Pharmaceutical grade
Form Lyophilized powder for reconstitution
Storage Refrigerate 2-8°C. Reconstituted: use within 3 hours.

Key Research References

  • Savarirayan R et al. "Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial." Lancet. 2020;396:684-92

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