BridgeBio Announces Infigratinib Is the First Ever Investigational Therapeutic Option for Achondroplasia to Be Awarded Breakthrough Therapy Designation by the FDA

Core Insights - The FDA granted Breakthrough Therapy Designation to infigratinib for children with achondroplasia, based on preliminary clinical evidence from the PROPEL 2 trial, indicating potential substantial improvement over existing therapies [1][2][3] - Infigratinib could become the first oral therapeutic option for children with achondroplasia if approved, which would significantly benefit the affected community [1][4] - The PROPEL 2 trial data showed a statistically significant increase in annualized height velocity (AHV) of +2.51 cm/year at Month 12 and +2.50 cm/year at Month 18, with p-values indicating strong statistical significance [1][3] Regulatory Designations - In addition to Breakthrough Therapy Designation, infigratinib has received Orphan Drug Designation, Fast Track Designation, and Rare Pediatric Disease Designation from the FDA [5] - The Breakthrough Therapy Designation is aimed at expediting the development and regulatory review process for drugs that show substantial improvement over existing therapies [2] Clinical Development - The ongoing PROPEL 3 Phase 3 registrational study of infigratinib is on schedule, with enrollment expected to be completed by the end of the year [4] - The company is committed to exploring the broader medical and functional impacts of infigratinib on achondroplasia and other skeletal dysplasias, addressing significant unmet needs [4]