Pemigatinib, a potent inhibitor of FGFRs for the treatment of cholangiocarcinoma

The prognosis of patients affected by cholangiocarcinoma is classically poor. Until recently, chemotherapeutic drugs were the only systemic treatment option available, leading to an overall survival lower than 1 year. In recent decades, different genetic alterations have been identified as playing a key role in the oncogenic signaling. A subgroup of intrahepatic cholangiocarcinoma is characterized byFGFRfamily mutations, more frequently represented by gene fusions ofFGFR2. Based on the results of FIGHT-202 trial, in April 2020 the US FDA approved the FGFR inhibitor pemigatinib in advanced previously treated cholangiocarcinoma patients withFGFR2rearrangements, opening the way to targeted therapy in this disease. This review summarizes the body of evidence about the efficacy of pemigatinib in cholangiocarcinoma.Lay abstract Cholangiocarcinoma is cancer that forms in the slender tubes bile ducts that carry the digestive fluid bile. This condition, also known as bile duct cancer, is a type of tumor that is very difficult to treat with common chemotherapy. Intrahepatic cholangiocarcinoma, those tumors occurring in the parts of the bile ducts within the liver, are frequently caused by alterations of a gene calledFGFR2. Pemigatinib is a novel potent drug that selectively inhibits the function of altered FGFR2 and recently demonstrated to be a valid treatment for patients affected by intrahepatic cholangiocarcinoma. Here, we present results about the efficacy of pemigatinib in this disease.