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.<Table Float="No" ID="Taba"> <tgroup cols="1"> <colspec align="left" colname="c1" colnum="1" /> <tbody> <row> <entry align="left" colname="c1"> What is Known: </entry> </row> <row> <entry align="left" colname="c1"> • Achondroplasia is a skeletal dysplasia caused by a mutation in the FGFR3 gene, which leads to inhibit endochondral ossification and bone growth. </entry> </row> <row> <entry align="left" colname="c1"> • Vosoritide is a recombinant C-type natriuretic peptide analogue designed to counteract overactive FGFR3 signaling and was approved by the FDA and EMA. </entry> </row> <row> <entry align="left" colname="c1"> What is New: </entry> </row> <row> <entry align="left" colname="c1"> • 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. </entry> </row> </tbody> </tgroup> </Table>

Efficacy and safety of vosoritide in children with achondroplasia: a systematic review and meta-analysis

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发表日期：

2026年4月24日

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Ghaidaa A. Alfaraj,
Hakeema A. Alfaraj,
Manar Adel,
Rahma B. AlGadeeb

摘要

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.