Drugdocs | Voxzogo®

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.

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.

Document Title	Year	Source
Voxzogo (vosoritide) Prescribing Information.	2023	BioMarin Pharmaceutical Inc., Novato, CA

Document Title	Year	Source
Burden and treatment of achondroplasia: a systematic literature review.	2023	Advances in Therapy
Assessment report: Voxzogo.	2021	EMA