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Phase 3 Trial of Oral Infigratinib in Children with Achondroplasia
Ravi Savarirayan1, Julie Hoover-Fong2, Melita Irving3
1Murdoch Children's Research Institute, Melbourne, VIC, Australia.
Insights
Infigratinib significantly increased annualized height velocity in children with achondroplasia over 52 weeks. This oral FGFR inhibitor showed promising results for improving growth in this genetic skeletal condition.
Area of Science:
- Genetics
- Pharmacology
- Pediatrics
Background:
- Achondroplasia is a genetic skeletal disorder caused by FGFR3 gene mutations.
- Infigratinib is an oral tyrosine kinase inhibitor targeting FGFR1-3 pathways involved in achondroplasia pathogenesis.
Purpose of the Study:
- To evaluate the efficacy and safety of infigratinib in children with achondroplasia.
- To assess the impact of infigratinib on height velocity and other growth parameters.
Main Methods:
- Phase 3, multicenter, double-blind, placebo-controlled trial.
- 114 children (3-17 years) randomized 2:1 to infigratinib (0.25 mg/kg/day) or placebo for 52 weeks.
- Primary endpoint: change in annualized height velocity; secondary endpoints: height z-score and upper-to-lower body segment ratio.
Main Results:
- Infigratinib showed a significant increase in annualized height velocity (1.74 cm/year) compared to placebo (P<0.001).
- Height z-score also significantly improved with infigratinib (0.32, P<0.001).
- Adverse events were frequent but generally mild; no treatment-related serious adverse events were reported.
Conclusions:
- Once-daily oral infigratinib significantly improved annualized height velocity in children with achondroplasia over 52 weeks.
- Infigratinib represents a potential therapeutic option for managing growth in achondroplasia.
Background:
Achondroplasia is a genetic skeletal condition caused by FGFR3 pathogenic variants. Infigratinib, an oral FGFR1-3 tyrosine kinase inhibitor, down-regulates key pathways in the pathogenesis of achondroplasia.
Methods:
In this phase 3, multicenter, double-blind, placebo-controlled trial, we randomly assigned children with achondroplasia (3 to 17 years of age) in a 2:1 ratio to receive infigratinib (at a dose of 0.25 mg per kilogram of body weight) or placebo once daily for 52 weeks. The primary end point was the change from baseline in the annualized height velocity in the infigratinib group as compared with the placebo group at week 52. Key secondary end points were the change from baseline in the height z score and in the upper-to-lower body segment ratio at week 52. The primary analysis evaluated the treatment effect at week 52 in the full analysis population, with missing data handled with a prespecified imputation approach.
Results:
In all, 114 patients underwent randomization: 75 patients to receive infigratinib (with 1 withdrawal before treatment) and 39 patients to receive placebo. The difference between infigratinib and placebo in the least-squares mean change from baseline to week 52 was 1.74 cm per year (95% confidence interval [CI], 1.31 to 2.17; P<0.001) for the annualized height velocity, 0.32 (96% CI, 0.23 to 0.41; P<0.001) for the height z score, and -0.02 (96% CI, -0.06 to 0.01) for the upper-to-lower body segment ratio. Adverse events occurred in 71 of 74 patients (96%) in the infigratinib group and in 37 of 39 patients (95%) in the placebo group; serious adverse events occurred in 4 of 74 patients (5%) and 1 of 39 patients (3%), respectively. No serious adverse events or adverse events leading to treatment discontinuation were considered by the investigator to be related to infigratinib or placebo.
Conclusions:
In children with achondroplasia, treatment with once-daily oral infigratinib for 52 weeks resulted in a significantly greater increase from baseline in the annualized height velocity than placebo. (Funded by BridgeBio Pharma; PROPEL 3 ClinicalTrials.gov number, NCT06164951; EudraCT number, 2023-506130-67.).
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