Oral Treatment Shows Promise for Children with Achondroplasia, NEJM Editorial Highlights

An editorial published in the New England Journal of Medicine (NEJM) on September 4, 2026, by Dr. Isidro B. Salusky of UCLA Health, highlights promising Phase 3 trial results for infigratinib, an oral treatment for children aged 3 to 17 with achondroplasia. The trial, originally published in June in NEJM, demonstrated that once-daily oral infigratinib for 52 weeks led to a significantly greater increase in baseline growth compared to placebo. This oral option is particularly suitable for infants, who benefit most from early pharmacological intervention.