Vosoritide (Voxzogo)

Peptide

Vosoritide is a 39-amino acid C-type natriuretic peptide (CNP) analog for achondroplasia. FDA-approved November 2021 - first pharmacological treatment for achondroplasia. Phase 3 trial (n=121): Significant increase in annualized growth velocity vs placebo at 52 weeks. Daily SC injection promotes endochondral bone growth by antagonizing FGFR3 downstream signaling. Approved for children 5+ years with open epiphyses.

Quick Answer

What it is

Vosoritide is a 39-amino acid C-type natriuretic peptide (CNP) analog for achondroplasia. FDA-approved November 2021 - first pharmacological treatment for achondroplasia.

Key findings

Grade A: Annualized Growth Velocity (Achondroplasia)
Grade A: Height Z-Score Improvement (Achondroplasia)
Grade A: Safety and Tolerability (Achondroplasia)

Safety

Phase 3 trial: Well tolerated with manageable adverse events.

⚠️ Research Notice

This peptide information is for educational and research purposes only. Peptides may not be FDA-approved for human use and may only be legally available for research purposes. Consult qualified healthcare professionals before considering any peptide compounds.

ℹ️ Quick Facts: Vosoritide (Voxzogo)

Quick Facts: Vosoritide (Voxzogo)

Best Evidence:Grade A
Conditions Studied:1
Research Outcomes:6
Grade A Findings:3
Grade B Findings:1
Key Effect:Achondroplasia

1 conditions · 6 outcomes

Detailed Outcomes

Annualized Growth Velocity

Phase 3 trial (n=121, ages 5-14): Vosoritide significantly increased annualized growth velocity vs placebo at 52 weeks. First disease-modifying treatment for achondroplasia. Led to FDA accelerated approval Nov 2021.

moderate↑Improves

Height Z-Score Improvement

Phase 3 trial: Significant improvement in height Z-scores vs placebo over 52 weeks. Increases in growth velocity sustained over treatment period. EMA approved for ages 2+ with open epiphyses.

moderate↑Improves

Safety and Tolerability

Phase 3 trial: Well tolerated with manageable adverse events. Most common: injection site reactions, transient blood pressure decreases. No serious safety signals. Approved via accelerated pathway.

moderate↑Improves

Long-Term Growth (Extension)

Open-label extension studies: Sustained growth velocity improvements over multiple years of treatment. Real-world data from multiple centers confirms efficacy. Treatment continues until epiphyseal closure.

moderate↑Improves