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

    Valeria Merz

    Digestive Molecular Clinical Oncology Research Unit, Università degli Studi di Verona, Verona, Italy

    Medical Oncology Unit, Santa Chiara Hospital, Trento, Italy

    ,
    Camilla Zecchetto

    Digestive Molecular Clinical Oncology Research Unit, Università degli Studi di Verona, Verona, Italy

    Experimental Cancer Medicine Unit, Azienda Ospedaliera Universitaria Integrata di Verona, Verona, Italy

    &
    Davide Melisi

    *Author for correspondence: Tel.: +39 045 812 8148;

    E-mail Address: davide.melisi@univr.it

    Digestive Molecular Clinical Oncology Research Unit, Università degli Studi di Verona, Verona, Italy

    Experimental Cancer Medicine Unit, Azienda Ospedaliera Universitaria Integrata di Verona, Verona, Italy

    Published Online:https://doi.org/10.2217/fon-2020-0726

    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 by FGFR family mutations, more frequently represented by gene fusions of FGFR2. 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 with FGFR2 rearrangements, 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 called FGFR2. 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.

    Papers of special note have been highlighted as: • of interest; •• of considerable interest

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