ROYAL CHILDREN'S HOSPITAL

Oral Infigratinib Shows Promise for Children with Achondroplasia

6 months ago

• A phase II study found that oral infigratinib was safe and effective in children with achondroplasia, showing a dose-dependent increase in height velocity. • The highest dose of infigratinib (0.25 mg/kg) resulted in a mean change from baseline of 2.5 cm per year in annualized height velocity at 18 months. • Infigratinib offers a potential alternative to the current standard treatment, vosoritide, which requires daily subcutaneous injections. • A phase 3, double-blind, placebo-controlled trial is underway to further evaluate infigratinib in a larger cohort of children with achondroplasia.