Drug updated on 12/11/2024
| Dosage Form | Injection (subcutaneous; 0.4 mg, 0.56 mg, 1.2 mg) |
| Drug Class | C type natriuretic peptide (CNP) analogs |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated to increase linear growth in pediatric patients with achondroplasia with open epiphyses.
Latest News
Summary
- This summary is based on the review of one systematic review/meta-analysis. [1]
- Vosoritide (Voxzogo) and growth hormone (GH) both conferred benefits for height or growth velocity in individuals with achondroplasia; however, the data for vosoritide were limited, and the long-term effects of GH therapy remain unclear.
- Limb lengthening also demonstrated benefits in height or growth velocity but was associated with complications, while vosoritide presented no specific safety concerns in the provided evidence.
- No significant differences in effectiveness were reported among various population types or subgroups in the comparison of vosoritide, GH, or limb lengthening.
- The systematic review focused on individuals with achondroplasia, highlighting the substantial burden of the condition on health-related quality of life and healthcare resource use. No specific subgroup analyses related to age, gender, or comorbid conditions were provided. The study noted benefits in height or growth velocity from vosoritide, growth hormone, and limb lengthening, though complications were associated with limb lengthening.
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 |