Details

Autor(en) / Beteiligte

• Rizzo, Alessandro
• Ricci, Angela Dalia
• Brandi, Giovanni

Titel

Pemigatinib: Hot topics behind the first approval of a targeted therapy in cholangiocarcinoma

Ist Teil von

• Cancer treatment and research communications, 2021, Vol.27, p.100337-100337, Article 100337

Ort / Verlag

England: Elsevier Ltd

Erscheinungsjahr

2021

Link zum Volltext

Quelle

Electronic Journals Library

Beschreibungen/Notizen

• •Unfortunately, although recent years have witnessed the advent of novel therapeutic options, patients with advanced cholangiocarcinoma (CCA) face a disappointing prognosis.•The fibroblast growth factor receptor (FGFR) inhibitor pemigatinib has reported remarkable results in the phase II FIGHT-202 trial.•Pemigatinib has received accelerated approval in April 2020 in the USA in previously treated CCA patients harboring FGFR2 fusions or other rearrangements.•However, several questions remain open, and further studies are warranted in this direction.•Results of ongoing clinical trials on pemigatinib and other selective FGFR inhibitors will provide further information regarding the role of these molecularly targeted therapies. Cholangiocarcinoma (CCA) includes a heterogeneous group of malignancies with limited treatment options. Despite recent advances in medical oncology, the prognosis of CCA patients with metastatic disease remains poor, with a median overall survival of less than a year. In the last decade, notable efforts have been made by the CCA medical community in an attempt to improve clinical outcomes of patients, with the development of molecularly targeted therapies in this setting. Among these treatments, the fibroblast growth factor receptor (FGFR) 2 inhibitor pemigatinib has received accelerated approval in April 2020 by the US Food and Drug Administration (FDA) in CCA patients harboring FGFR2 gene fusions or other rearrangements, on the basis of the results of the FIGHT-202 trial, and thus, representing the first molecularly targeted therapy to be approved for the treatment of CCA. However, several issues remain, including the emergence of polyclonal mutations determining resistance to pemigatinib, the identification of biomarkers predictive of response, and the knowledge gaps regarding the role of other FGFR gene aberrations. This review aims to provide an overview of recent development of pemigatinib, especially focusing on the results of the pivotal FIGHT-202 trial, the approval of this FGFR inhibitor, and the future challenges concerning the use of FGFR-directed treatments in CCA patients.