Multinational phase 2 study of futibatinib for unresectable or metastatic FGFR2-rearranged intrahepatic cholangiocarcinoma

Alterations in FGFR2 have emerged as promising drug targets for intrahepatic cholangiocarcinoma (ICC). Futibatinib is a next-generation, covalently binding FGFR1-4 inhibitor. The multinational phase 2 FOENIX-CCA2 trial evaluated futibatinib for unresectable or metastatic FGFR2-rearranged ICC after at least one previous line of systematic therapy. Response was seen in 42% of the patients (95% CI 32-52) with median duration of response 9.7 months. At median follow-up of 17.1 months, the median progression-free survival was 9.0 months and median overall survival was 21.7 months. Treatment-related adverse events led to permanent discontinuation of futibatinib in 2% of the patients. The authors conclude that in previously treated patients with FGFR2-rearranged ICC, use of futibatinib led to measurable clinical benefit (visual abstract).