Systematic review and meta-analysis of vosoritide in children with achondroplasia shows growth gains and injection site reactions

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European journal of pediatrics Published April 24, 2026 Medically reviewed May 24, 2026 Study authors: Alfaraj Ghaidaa A, Alfaraj Hakeema A, Adel Manar, AlGadeeb Rahma B PubMed ↗ DOI ↗ By Dr. Sofia Müller, MD · Lifespan & Whole-Person Care

Key Takeaway

Consider vosoritide for achondroplasia; note 51% injection site reactions and need for longer-term safety data.

This systematic review and single-arm meta-analysis synthesized data from 13 studies evaluating vosoritide at the approved dose of 15 μg/kg/day in children with genetically confirmed achondroplasia. The analysis covered a follow-up period of 12 months and focused on growth metrics rather than comparative efficacy against a placebo or other active comparator, as the comparator was not reported.

The primary outcomes showed that vosoritide was associated with an annualized growth velocity of 5.72 cm/year, with a 95% CI of 5.51-5.94. Additionally, the analysis reported a height Z-score improvement of 0.28, with a 95% CI of 0.16-0.4. These findings indicate increased growth velocity and height gain relative to baseline expectations in this population.

Safety data indicated a high incidence of mild to moderate adverse events. Specifically, injection site reactions occurred in 51% of patients, and gastrointestinal symptoms occurred in 50%. Serious adverse events and discontinuations were not reported in the provided data. The authors note that larger, longer-term studies are necessary to confirm the treatment's safety and efficacy.

The practice relevance is limited by the observational nature of the meta-analysis and the lack of long-term safety data. Clinicians should interpret these pooled effect sizes as associations rather than definitive causal claims, particularly given the absence of a control group in the underlying single-arm design.

Study Details

Study typeMeta analysis

EvidenceLevel 1

Follow-up12.0 mo

PublishedApr 2026

PMID42026358

View Original Abstract ↓

UNLABELLED: Achondroplasia is caused by a gain-of-function mutation in the FGFR3 gene. Vosoritide activates the NPR-B receptor to inhibit the overactive FGFR3 signaling pathway. We aim to pool the efficacy and safety outcomes of vosoritide in children with genetically confirmed achondroplasia who are receiving the approved dose of 15 μg/kg/day. We searched five databases up to February 10, 2026. A systematic review and single-arm meta-analysis were conducted in accordance with the PRISMA guidelines. The primary outcomes were annualized growth velocity (AGV), height gain, and change in height Z-score. Three quality assessment tools were used to assess different study designs of the included studies. All analyses were conducted using OnlineMeta V1.1. Thirteen studies on vosoritide treatment in children with achondroplasia were included, comprising randomized controlled trials, cohort studies, case reports, and case series. A meta-analysis showed that vosoritide was associated with an AGV of 5.72 cm/year (95% CI: 5.51-5.94) at 12 months. The mean height Z-score improvement at 12 months after sensitivity analysis was 0.28 (95% CI: 0.16-0.4), with no significant difference between sexes. Overall, the most common adverse events were injection site reactions (51%) and gastrointestinal symptoms (50%). CONCLUSION: One-year treatment with vosoritide is associated with increased growth velocity, height gain, and a modest improvement in height Z-score, accompanied by a high incidence of mild to moderate adverse events. Larger, longer-term studies are necessary to confirm the treatment's safety and efficacy. WHAT IS KNOWN: • Achondroplasia is a skeletal dysplasia caused by a mutation in the FGFR3 gene, which leads to inhibit endochondral ossification and bone growth. • Vosoritide is a recombinant C-type natriuretic peptide analogue designed to counteract overactive FGFR3 signaling and was approved by the FDA and EMA. WHAT IS NEW: • One-year treatment with vosoritide in achondroplasia children is associated with increased growth velocity and improvement in height Z-score with reported minor adverse events.