Infigratinib for cholangiocarcinoma.

Abstract

Cholangiocarcinoma (CCA) is a serious and often fatal cancer of the bile ducts of the liver with a 5-year survival rate of 5-15%. At the time of diagnosis, most patients present with advanced or metastatic CCA, which is an aggressive malignancy with a poor prognosis. The standard of care for patients with locally advanced or metastatic CCA includes systemic chemotherapy with gemcitabine and cisplatin. Recently, research in the molecular basis of cancer has led to the discovery of underlying gene alterations, allowing the development of targeted therapies. Here we provide a review of infigratinib, which is an oral small-molecule tyrosine kinase inhibitor targeting fibroblast growth factor receptor (FGFR). On the basis of positive phase II efficacy data, infigratinib received accelerated approval from the U.S. Food and Drug Administration (FDA) for adults with previously treated, unresectable locally advanced or metastatic CCA with an FGFR2 fusion or other rearrangement.