BridgeBio Reports Positive Phase 3 Topline Results for Oral Infigratinib with the First Statistically Significant Improvements in Body Proportionality in Achondroplasia

Core Insights - BridgeBio Pharma announced positive topline results from the PROPEL 3 study, demonstrating the efficacy of oral infigratinib in children with achondroplasia, achieving significant improvements in height velocity and body proportionality [1][2][3] Study Results - PROPEL 3 met its primary endpoint with a statistically significant change from baseline in annualized height velocity (AHV) at Week 52, showing a mean treatment difference of +2.10 cm/year compared to placebo [1][2] - The study also achieved key secondary endpoints, including a significant improvement in height Z-score with an LS mean increase of +0.41 SD [1][3] - Infigratinib demonstrated the first statistically significant improvement in body proportionality in children younger than 8 years old, with an LS mean treatment difference of -0.05 against placebo [1][6] Safety Profile - Oral infigratinib was well tolerated, with no discontinuations or serious adverse events related to the study drug, and only mild, transient cases of hyperphosphatemia reported [1][6] Regulatory Plans - The company plans to submit a New Drug Application (NDA) and Marketing Authorization Application (MAA) for infigratinib in the second half of 2026, marking it as the only therapeutic option in development for achondroplasia with Breakthrough Therapy Designation from the FDA [1][3] Future Development - BridgeBio intends to accelerate the development of infigratinib for hypochondroplasia and is currently enrolling participants for the Phase 3 trial [1][3]