BridgeBio Pharma Inc. (NASDAQ: BBIO) reported that oral infigratinib produced improvements in respiratory outcomes and growth metrics during its Phase 3 PROPEL 3 study in children with achondroplasia, data presented at the Annual European Society for Paediatric Endocrinology Meeting in Marseille, France.
The mean total Apnea-Hypopnea Index rose 10.4% in the infigratinib group versus a 49.2% increase on placebo. Among children younger than 8, mean total AHI remained essentially unchanged with infigratinib while rising 63.2% on placebo.
The estimated annualized rate of otitis media events fell 38% in the infigratinib group compared with placebo, with a 47% reduction seen in children under 8.
Body composition measures also shifted favorably. Mean change from baseline in BMI was 0.50 kg/m2 with infigratinib versus 0.93 kg/m2 on placebo. Lean body mass increased 1.77 kg with the drug compared to 1.58 kg on placebo. Children treated for up to three years showed a height Z-score improvement of +0.92 SD at Year 3, alongside a -0.15 change in upper-to-lower body segment ratio.
BridgeBio said no new safety signals were identified.
The company has submitted a New Drug Application to the FDA for infigratinib in achondroplasia and anticipates a U.S. launch in mid-2027. A Marketing Authorization Application to the European Medicines Agency is planned for the fourth quarter of 2026. Infigratinib holds Breakthrough Therapy, Orphan Drug, Fast Track and Rare Pediatric Disease designations from the FDA.
Achondroplasia affects approximately 55,000 people in the U.S. and European Union, including up to 10,000 children and adolescents with open growth plates. The 52-week PROPEL 3 study is part of the broader PROPEL program, in which some children have received treatment for up to three years.
