Peptide
Vosoritide (Voxzogo)
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.
- Updated
- How we grade
- 26 studies cited
Research notice. This page is for education and research. Peptides may not be FDA-approved for human use and may only be legally available for research. Talk to a qualified clinician before considering any peptide.
- Best evidence
- Grade A
- Conditions studied
- 1
- Outcomes
- 6
- Graded outcomes
- 3102
Evidence by condition
- Strong
- Moderate
- Limited
- Very limited
General
Key findings
- Annualized Growth VelocityImproves (the measure goes up)
- Height Z-Score ImprovementImproves (the measure goes up)
- Safety and TolerabilityImproves (the measure goes up)
Safety notes in the studies
- Phase 3 trial: Well tolerated with manageable adverse events.
Studies cited
26 studies from PubMed
- Real-world safety and age-dependent effectiveness of vosoritide in achondroplasia: A single-center retrospective analysis of transition from growth hormone to vosoritide.
- Exploring adverse events associated with vosoritide monotherapy: Insights from the FDA Adverse Event Reporting System.
- Phase 2 Trial of Vosoritide Use in Patients with Hypochondroplasia: A Pharmacokinetic/Pharmacodynamic Analysis.
- Real-World Safety and Effectiveness of Vosoritide in Children with Achondroplasia: French Early Access Program.
- Real-World Safety and Effectiveness of Vosoritide in Achondroplasia: Results from a Single Center in Portugal.
- Three-dimensional craniofacial imaging in children with achondroplasia treated with vosoritide.
- Effect of Vosoritide therapy on IGF-I and Endogenous C-type Natriuretic Peptide in Hypochondroplasia.
- Sustained growth-promoting effects of vosoritide in children with achondroplasia from an ongoing phase 3 extension study.
- [An update review of advances in the treatment of achondroplasia].
- Sleep-disordered breathing in children with achondroplasia assessed by polysomnography: a retrospective chart review.
- Vosoritide (Voxzogo) for Achondroplasia: A Review of Clinical and Real-World Evidence.
- Real-world outcomes of vosoritide in achondroplasia: A systematic review and meta-analysis of multinational clinical evidence.
- Two Cases of Cardiovascular Adverse Events Following Subcutaneous Vosoritide Injection in Early Infancy.
- Progress in managing children with achondroplasia.
- Vosoritide therapy in children with achondroplasia aged 3-59 months: a multinational, randomised, double-blind, placebo-controlled, phase 2 trial.
- Clinical outcomes and medical management of achondroplasia in Japanese children: A retrospective medical record review of clinical data.
- Vosoritide treatment for children with hypochondroplasia: a phase 2 trial.
- Assessment of the efficacy of vosoritide therapy in children with achondroplasia in clinical trials.
- Burden and Treatment of Achondroplasia: A Systematic Literature Review.
- Literature review and expert opinion on the impact of achondroplasia on medical complications and health-related quality of life and expectations for long-term impact of vosoritide: a modified Delphi study.
- Rationale, design, and methods of a randomized, controlled, open-label clinical trial with open-label extension to investigate the safety of vosoritide in infants, and young children with achondroplasia at risk of requiring cervicomedullary decompression surgery.
- Advantages and Disadvantages of Different Treatment Methods in Achondroplasia: A Review.
- Safe and persistent growth-promoting effects of vosoritide in children with achondroplasia: 2-year results from an open-label, phase 3 extension study.
- Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial.
- C-Type Natriuretic Peptide Analogue Therapy in Children with Achondroplasia
- Evaluation of the therapeutic potential of a CNP analog in a Fgfr3 mouse model recapitulating achondroplasia