Drug updated on 5/17/2024
Dosage Form | Injection (subcutaneous; 0.4 mg, 0.56 mg, 1.2 mg) |
Drug Class | C type natriuretic peptide analogs |
Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated to increase linear growth in pediatric patients with achondroplasia who are 5 years of age and older with open epiphyses.
Latest News
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Summary
- Vosoritide (Voxzogo) is indicated to increase linear growth in pediatric patients with achondroplasia who are 5 years of age and older with open epiphyses.
- The information was derived from a single systematic literature review/meta-analysis study, which compared Voxzogo's safety and effectiveness against other treatments such as growth hormone therapy and limb lengthening surgery.
- Among the treatments discussed for height or growth velocity issues in patients with achondroplasia, vosoritide has shown benefits. However, data supporting its use come from limited studies, suggesting that more research is needed to fully understand its long-term effects and overall safety profile.
- Growth Hormone therapy also shows potential, but there are concerns about unclear long-term effects, requiring careful consideration due to uncertainties regarding sustained benefits and potential side-effects.
- Limb Lengthening offers benefits for growth but comes along with complications, making it a less desirable option due to associated risks, especially among families considering this surgical option for their children suffering from achondroplasia.
- While the study does not provide detailed outcomes based on population subgroups within achondroplasia, it indicates that the primary focus of treatment considerations lies on pediatric patients having open epiphyses.
Product Monograph / Prescribing Information
Document Title | Year | Source |
---|---|---|
Voxzogo (vosoritide) Prescribing Information. | 2023 | BioMarin Pharmaceutical Inc., Novato, CA |
Systematic Reviews / Meta-Analyses
Document Title | Year | Source |
---|---|---|
Burden and treatment of achondroplasia: a systematic literature review. | 2023 | Advances in Therapy |
Clinical Practice Guidelines
Document Title | Year | Source |
---|---|---|
Emerging therapies for the treatment of rare pediatric bone disorders. | 2022 | Frontiers in Pediatrics |