Phase 3 Trial of Oral Infigratinib in Children with Achondroplasia.

In children with achondroplasia, 52 weeks of oral infigratinib increased annualized height velocity by 1.74 cm compared to placebo, showing significant growth benefits.

• Why it matters: Achondroplasia, caused by FGFR3 variants, leads to short stature and skeletal abnormalities, with limited effective treatments. Addressing this gap could improve growth outcomes and quality of life for affected children.
• What they did: A phase 3, multicenter, double-blind, placebo-controlled trial enrolled 114 children aged 3 to 17, randomly assigning them in a 2:1 ratio to receive daily infigratinib or placebo for one year, measuring height velocity and other growth parameters.
• The result: Treatment with infigratinib significantly improved height velocity and height z scores without serious adverse events related to the drug, supporting its potential as a safe, effective therapy for achondroplasia in children.