Phase 3 Trial of Oral Infigratinib in Children with Achondroplasia.

N Engl J MedJun 28, 2026 (epub)
Clinical ResearchMedical GeneticsPediatrics

Ravi Savarirayan, Julie Hoover-Fong, Melita Irving et al.

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In 30 seconds

This phase 3, multicenter, double-blind, placebo-controlled trial evaluated the efficacy of oral infigratinib in children aged 3 to 17 with achondroplasia. The study found that infigratinib significantly increased annualized height velocity by 1.74 cm per year compared to placebo after 52 weeks.

Key findings

  • Infigratinib increased annualized height velocity by 1.74 cm per year compared to placebo (P<0.001).
  • The height z score improved by 0.32 in the infigratinib group (P<0.001).
  • Serious adverse events occurred in 5% of the infigratinib group and 3% of the placebo group.

Why it matters

The findings suggest that infigratinib may provide a new therapeutic option for improving growth in children with achondroplasia, a condition that can significantly impact quality of life and physical development.

Source

PubMed · PMID 42370681

Published in N Engl J Med. This summary was written by xxcode from the publication's abstract and metadata. It is not peer reviewed and is not a substitute for the original article. For clinical decisions, review the original publication.

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