Background and objectives Achondroplasia, a rare autosomal dominant disorder caused by FGFR3 mutation, is treated with vosoritide, a C-type natriuretic peptide analogue. This review aimed to assess vosoritide’s efficacy and safety to guide clinical practice and healthcare policy decisions. Methods Advanced search was performed on three databases. The included articles underwent screening, data extraction, and quality evaluation, culminating in a qualitative synthesis. The protocol was registered on PROSPERO (CRD42024541795). Results Out of 752 records screened, six were incorporated in this systematic review. The trials included daily injections of vosoritide (2.5-30 µg/kg) in phase II study and at 15 µg/kg in all other studies to patients with achondroplasia aged 3 months to 18 years. Safety assessments were done for all patients (n=156) having received vosoritide. Efficacy was assessed using annualised growth velocity and height Z score as the primary outcomes andserum collagen X-marker concentrations, bone age progression, and serum immunogenicity as secondary outcomes. Overall, the articles demonstrated good quality with minimal bias. Interpretation and conclusions Vosoritide showed significant improvement in the annualised growth velocity, height z score and standing height in patients with achondroplasia aged 3 months to 18 years as compared to placebo. The safety assessment recorded adverse events in all patients (n=156) enrolled, usually mild (grade 1), self-limiting and limited to local injection site reactions. Studies with longer duration (till puberty), a large sample size and its effect on medical complications, are required to establish its effectiveness in the patients of achondroplasia.
Pahuja et al. (Sat,) studied this question.