A phase 3 trial published in the NEJM found that infigratinib, a once-daily oral medication, significantly boosted height velocity in children with achondroplasia compared to placebo. Kids on the drug grew 1.74 cm per year more than those on placebo — with a strong safety profile. It could become the first oral therapy approved for this genetic condition.
A phase 3 trial (PROPEL 3) published in The New England Journal of Medicine has shown that infigratinib — a once-daily oral tyrosine kinase inhibitor developed by BridgeBio Pharma — significantly improved height velocity in children and adolescents with achondroplasia, the most common form of dwarfism. The trial enrolled 114 children aged 3–17 years, randomly assigned 2:1 to infigratinib or placebo for one year.
Children on infigratinib grew 1.74 cm per year more than those on placebo, and their height z-scores improved meaningfully. Researchers are optimistic that sustained treatment could help children approach more typical adult height ranges. The drug also showed early signals of improving body proportionality — a key quality-of-life concern beyond just height. BridgeBio plans to file for regulatory approval in the second half of 2026.
By the Numbers:
Why it matters: Achondroplasia affects roughly 1 in 25,000 births and carries significant skeletal and functional complications. Current approved therapies require daily injections. If approved, infigratinib would be the first oral treatment for achondroplasia — a potentially transformative option for families and clinicians alike.
