Vosoritide

(Voxzogo®)

Voxzogo®

Drug updated on 5/17/2024

Dosage FormInjection (subcutaneous; 0.4 mg, 0.56 mg, 1.2 mg)
Drug ClassC 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
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  • 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 TitleYearSource
Voxzogo (vosoritide) Prescribing Information.2023BioMarin Pharmaceutical Inc., Novato, CA

Systematic Reviews / Meta-Analyses

Document TitleYearSource
Burden and treatment of achondroplasia: a systematic literature review.2023Advances in Therapy

Clinical Practice Guidelines

Document TitleYearSource
Emerging therapies for the treatment of rare pediatric bone disorders.2022Frontiers in Pediatrics