Pemigatinib (Pemazyre)

General information

Class/mechanism from the NCI Drug Dictionary: An orally bioavailable inhibitor of the fibroblast growth factor receptor (FGFR) types 1, 2, and 3 (FGFR1/2/3), with potential antineoplastic activity. Pemigatinib binds to and inhibits FGFR1/2/3, which may result in the inhibition of FGFR1/2/3-related signal transduction pathways. This inhibits proliferation in FGFR1/2/3-overexpressing tumor cells.

Diseases for which it is used

• Cholangiocarcinoma
• FGFR1+ myeloid/lymphoid neoplasms (site-agnostic)

History of changes in FDA indication

• 2020-04-17: Granted accelerated approval for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement. (Based on FIGHT-202)
• 2022-08-26: Approved for adults with relapsed or refractory myeloid/lymphoid neoplasms (MLNs) with fibroblast growth factor receptor 1 (FGFR1) rearrangement. (Based on FIGHT-203)

History of changes in EMA indication

• 2021-03-26: Initial conditional authorization as Pemazyre.

History of changes in Health Canada indication

• 2021-09-17: Initial notice of compliance with conditions for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement.

History of changes in PMDA indication

• 2021-03-21: Newly indicated for the treatment of unresectable FGFR2 fusion gene-positive biliary tract cancer that has progressed after cancer chemotherapy.

Also known as

• Code name: INCB-054828
• Brand name: Pemazyre