Phase 3 trial of oral infigratinib shows significant growth improvement in children with achondroplasia, published in NEJM.

BridgeBio Pharma announced that the Phase 3 PROPEL 3 trial of oral infigratinib in children with achondroplasia demonstrated significant improvements in height velocity, body proportionality, and arm span compared to placebo. These results, published in the New England Journal of Medicine, mark the first Phase 3 achondroplasia study with statistically significant placebo-controlled outcomes. The drug was well tolerated with no serious adverse events, and BridgeBio plans to submit regulatory applications to the FDA and EMA in 2026, aiming for a U.S. launch by mid-2027. This therapy targets the FGFR3 mutation causing achondroplasia and could become the first approved oral treatment for this condition.