U.S. Department of Health & Human Services Divider Arrow National Institutes of Health Divider Arrow NCATS

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Pemigatinib, an oral kinases inhibitor, was approved under the brand name PEMAZYRE for the treatment of adults with previously treated, unresectable locally advanced, or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion. 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. The drug inhibited FGFR1-3 phosphorylation and signaling and decreased cell viability in cancer cell lines with activating FGFR amplification and fusions that resulted in constitutive activation of FGFR signaling.