Drug updated on 3/28/2024
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.
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.
- A systematic literature review was conducted, which included a total of one document that discussed the efficacy, safety, health-related quality of life (HRQoL), healthcare resource use (HCRU), costs and economic evaluation data related to vosoritide for treating achondroplasia.
- The study found that vosoritide conferred benefits for height or growth velocity in children affected by achondroplasia; however, these findings were based on a limited number of studies.
- Compared to other treatments such as growth hormone therapy and limb lengthening procedures, vosoritide showed promising results but long-term effects remain unclear due to lack of sufficient data.
- The research also highlighted substantial HRQoL burden associated with emotional wellbeing issues among individuals suffering from achondroplasia along with significant HCRU/cost-related burdens particularly concerning hospitalization costs and resource use.
- There exists an evidence gap regarding utility and cost-effectiveness data for both the condition itself -achondroplasia- as well as its treatment options including vosoritide; this necessitates further research into these areas according to the reviewed document's conclusions.
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 |
Assessment report: Voxzogo. | 2021 | EMA |