Pemigatinib is a small molecule kinase inhibitor with antitumour activity. It works by inhibiting fibroblast growth factor receptors (FGFRs), which are receptor tyrosine kinases that activate signalling pathways in tumour cells. FGFRs gained attention as potential therapeutic targets in selected cancers, as FGFR gene alterations were observed in a wide variety of cancers including those of the urinary bladder, breast, ovary, prostate, endometrium, lung, and stomach. Deregulated FGFR signalling pathway can lead the development of oncogenes and tumour-promoting physiological processes, such as cancer cell proliferation, enhanced angiogenesis, and evasion of cell death. In April 2020, pemigatinib was approved by the FDA for the treatment of unresectable locally advanced or metastatic cholangiocarcinoma in previously treated adult patients with a fibroblast growth factor receptor 2 (FGFR2) gene fusion or other rearrangements as detected by an FDA-approved test. Cholangiocarcinoma is the most common primary malignancy affecting the biliary tract and the second most common primary hepatic malignancy. This malignancy accounts for 15% to 20% of primary hepatobiliary malignancies, which account for 13% of overall cancer-related global mortality. With increasing research on the pathogenesis of cholangiocarcinoma and potential therapeutic targets for anticancer drug treatment, recent studies show that up to 45% of patients with intrahepatic cholangiocarcinoma exhibited gene rearrangements resulting in oncogenic fibroblast growth factor 2 (FGFR2) fusion proteins. The FDA-approved indication for pemigatinib was granted under accelerated approval based on the overall response rate and duration of response in pre-marketing clinical trials. Pemigatinib is marketed under the brand name Pemazyre, and it is available as oral tablets.

Pemigatinib is indicated for the treatment of unresectable locally advanced or metastatic cholangiocarcinoma in previously-treated adult patients with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test. It is also indicated for the treatment of adults with relapsed or refractory myeloid/lymphoid neoplasms (MLNs) with FGFR1 rearrangement.

• Refractory Myeloid/Lymphoid Neoplasms With FGFR1 Rearrangement
• Relapsed Myeloid/Lymphoid Neoplasms With FGFR1 Rearrangement
• Unresectable, locally advanced Cholangiocarcinomas
• Unresectable, metastatic Cholangiocarcinomas