Condition
Achondroplasia
Achondroplasia is a genetic condition in which a change in the FGFR3 gene overactivates a signal that slows cartilage turning into bone, leading to shorter limbs. Vosoritide, a medicine rather than a supplement, has the strongest evidence: in a trial of 121 children, it increased yearly growth speed compared with placebo. That trial ran 52 weeks in ages 5 to 14, so results mostly apply to children in that range.
Sources: PMID 38556428; PMID 36566254
- Updated
- How we grade
- 32 studies cited
- Supplements studied
- 0
- Medicines and peptides
- 1
- Graded outcomes
- 4
Prescription medicines and peptides studied, for context
| Grade | Outcome | Effect | Size | Studies | People | Studies list |
|---|---|---|---|---|---|---|
| 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. | Improves (the measure goes up) | Moderate effect | 22 studies | |||
Studies that measured annualized growth velocity | ||||||
| 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. | Improves (the measure goes up) | Moderate effect | 20 studies | |||
Studies that measured height z-score improvement | ||||||
| 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. | Improves (the measure goes up) | Moderate effect | 20 studies | |||
Studies that measured safety and tolerability | ||||||
| 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. | Improves (the measure goes up) | Moderate effect | 23 studies | |||
Studies that measured long-term growth (extension) | ||||||
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
32 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.
- International consensus guidelines on the implementation and monitoring of vosoritide therapy in individuals with achondroplasia
- Vosoritide approved for treatment of linear growth in pediatric patients with achondroplasia: A therapeutics bulletin of the American College of Medical Genetics and Genomics (ACMG)
- Vosoritide treatment for children with hypochondroplasia: a phase 2 trial
- 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.
- Growth outcomes and safety of vosoritide in children with achondroplasia under 5 years of age: A prospective study
- Burden and Treatment of Achondroplasia: A Systematic Literature Review.
- Vosoritide: A Review in Achondroplasia
- 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
- 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