Efficacy and safety of Vosoritide in achondroplasia: A systematic review and meta-analysis

source extracted confidence: high 2026-04-09
#humans #achondroplasia #natriuretic-peptide-c-type #adolescent #child

Efficacy and safety of Vosoritide in achondroplasia: A systematic review and meta-analysis

Pahuja et al., 2026 | Indian J Med Res | Meta Analysis

Citation

Pahuja Monika, Prakash Chander, ... Chatterjee Nabendu Sekhar. Efficacy and safety of Vosoritide in achondroplasia: A systematic review and meta-analysis. Indian J Med Res. 2026-Jan;163(1):78-87. doi:10.25259/IJMR_2087_2025

Abstract

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.

Key Findings

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.

Outcomes Measured

  • Requires manual extraction

Population

Field Value
Population achondroplasia aged 3 months
Sample Size 156
Age Range See abstract
Condition See abstract

MeSH Terms

  • Humans
  • Achondroplasia
  • Natriuretic Peptide, C-Type
  • Adolescent
  • Child
  • Receptor, Fibroblast Growth Factor, Type 3
  • Child, Preschool
  • Infant
  • Mutation

Evidence Classification

  • Level: Meta Analysis
  • Publication Types: Journal Article, Systematic Review, Meta-Analysis, Review
  • Vertical: collagen

Provenance

Source extracted via PubMed E-utilities API on 2026-04-09