Infigratinib Improves Growth Velocity in Children with Achondroplasia: Study
USA: In children with achondroplasia, once-daily oral infigratinib treatment for 52 weeks significantly increased annualized height velocity compared with placebo, highlighting its potential as an effective therapy to improve growth in affected children.
The findings were reported in a phase 3, multicenter, double-blind, placebo-controlled trial published in The New England Journal of Medicine. The study evaluated the efficacy and safety of infigratinib, an oral fibroblast growth factor receptor (FGFR) 1–3 tyrosine kinase inhibitor, in children with achondroplasia.
Achondroplasia is a genetic skeletal disorder caused by pathogenic variants in the FGFR3 gene. These genetic changes disrupt signaling involved in bone growth and development, resulting in characteristic short stature and skeletal abnormalities. Infigratinib is designed to modulate FGFR signaling pathways implicated in the condition.
The trial enrolled 114 children aged 3 to 17 years with achondroplasia. Participants were randomly assigned in a 2:1 ratio to receive either infigratinib at a dose of 0.25 mg/kg once daily or placebo for 52 weeks. Of the participants, 75 were assigned to infigratinib, with one withdrawing before treatment, while 39 received placebo.
The primary endpoint was the change from baseline in annualized height velocity at week 52. Researchers also assessed changes in height z score and the upper-to-lower body segment ratio as key secondary outcomes.
The trial revealed the following findings:
- After 52 weeks, infigratinib significantly increased annualized height velocity compared with placebo, with a least-squares mean difference of 1.74 cm/year.
- Infigratinib also significantly improved the height z score, with a between-group difference of 0.32.
- No significant difference was observed in the upper-to-lower body segment ratio between groups (difference, −0.02).
- Adverse events occurred in 96% of patients receiving infigratinib (71/74) and 95% receiving placebo (37/39).
- Serious adverse events were reported in 5% of the infigratinib group (4/74) and 3% of the placebo group (1/39).
- Investigators did not consider any serious adverse events or adverse events resulting in treatment discontinuation to be related to infigratinib or placebo.
The researchers concluded that 52 weeks of once-daily oral infigratinib significantly increased annualized height velocity compared with placebo in children with achondroplasia. The findings support further consideration of FGFR-targeted treatment as a potential approach to improving linear growth in children affected by this genetic skeletal disorder.
Reference:
Savarirayan R, Hoover-Fong J, Irving M, Arundel P, de Bergua JM, Campeau PM, Candler T, Cocanougher BT, Cormier-Daire V, Edouard T, Fredwall SO, Harmatz P, Hoernschemeyer D, Irgens HU, Jamuar S, Kannu P, Legare JM, Leiva-Gea A, McDevitt H, Onesimo R, Phillips J 3rd, Del Pino M, Robinson ME, Rossi M, Skae M, Ward LM, White KK, Schmidt J, Lystig T, Salvatici A, Bai Y, Butler PW, van Veenhuyzen D, Rogoff D. Phase 3 Trial of Oral Infigratinib in Children with Achondroplasia. N Engl J Med. 2026 Sep 3;395(9):859-869. doi: 10.1056/NEJMoa2604565. Epub 2026 Jun 28. PMID: 42370681.
Next Story