Oral Infigratinib in Children with Achondroplasia

This editorial discusses the results of a phase 2 trial evaluating oral infigratinib in children with achondroplasia. Infigratinib, a selective FGFR1–3 inhibitor, targets the excessive signaling caused by FGFR3 mutations that impair chondrocyte proliferation and skeletal growth. The trial demonstrated dose-dependent increases in annualized height velocity, with the highest dose cohort achieving a 2.50 cm/year gain over baseline. Adverse events were mostly mild to moderate. Infigratinib’s oral formulation offers an administration advantage over subcutaneous vosoritide. However, long-term effects on pubertal growth, adult bone health, and body proportions remain unknown. The findings support infigratinib as a promising targeted therapy pending further validation in phase 3 trials.

This editorial discusses the results of a phase 2 trial evaluating oral infigratinib in children with achondroplasia. Infigratinib, a selective FGFR1–3 inhibitor, targets the excessive signaling caused by FGFR3 mutations that impair chondrocyte proliferation and skeletal growth. The trial demonstrated dose-dependent increases in annualized height velocity, with the highest dose cohort achieving a 2.50 cm/year gain over baseline. Adverse events were mostly mild to moderate. Infigratinib’s oral formulation offers an administration advantage over subcutaneous vosoritide. However, long-term effects on pubertal growth, adult bone health, and body proportions remain unknown. The findings support infigratinib as a promising targeted therapy pending further validation in phase 3 trials.