BridgeBio Pharma-supported oral infigratinib delivered statistically significant improvements versus placebo in a Phase 3 trial in children with achondroplasia, according to a June 28 publication at NEJM.org. In the PROPEL 3 study (NCT06164951), infigratinib increased annualized height velocity by 1.74 cm per year versus placebo at week 52 (P<0.001) and improved height z score. The drug’s adverse-event profile showed no investigator-attributed serious adverse events in either arm, with serious events reported in 5% of treated patients and 3% of placebo recipients. Investigators also reported no serious adverse events or adverse events leading to treatment discontinuation deemed related to infigratinib. For biotech and rare-disease stakeholders, the trial provides pivotal confirmatory efficacy data for a daily oral FGFR1–3 tyrosine kinase inhibitor approach, supporting future regulatory and labeling discussions for pediatric growth outcomes.