Futibatinib

Futibatinib is an inhibitor of Fibroblast Growth Factor receptor (FGFR), which comprises a group of receptor tyrosine kinases that play a key role in cell proliferation, differentiation, migration, and survival. FGFR was investigated in oncology as a therapeutic target, as FGFR genomic aberrations and dysregulated FGFR signalling pathways are observed in some cancers such as cholangiocarcinoma and urothelial malignancies.

As a novel inhibitor of FGFR, futibatinib was first approved by the FDA in September 2022 to treat different types of intrahepatic cholangiocarcinoma. On July 4, 2023, the European Commission granted Conditional Marketing Authorization for futibatinib for the treatment of cholangiocarcinoma.

Futibatinib is indicated to treat adults with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma harbouring fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangements. In Europe, it is indicated in patients whose disease has progressed after at least one prior line of systemic therapy.

Futibatinib is approved in the US under accelerated approval and in Europe under conditional marketing authorization. This currently approved indication is subject to change, as it may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).