Cholangiocarcinoma Medication

Updated: Oct 11, 2022
  • Author: Peter E Darwin, MD; Chief Editor: N Joseph Espat, MD, MS, FACS  more...
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Medication

Medication Summary

Few patients are diagnosed with early-stage resectable tumors. Guidelines for unresectable or metastatic disease include various chemotherapy regimens. Targeted agents are available as second-line therapy for cholangiocarcinoma with specific driver mutations, including fibroblast growth factor receptor 2 (FGFR2)–selective tyrosine kinase inhibitors for advanced cholangiocarcinoma harboring an FGFR2 gene fusion or rearrangement, and an isocitrate dehydrogenase 1 (IDH1) inhibitor for IDH1-mutated cholangiocarcinoma.

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FGFR Inhibitors

Class Summary

Inhibition of FGFR disrupts tumor cell proliferation, survival, migration, and angiogenesis.

Pemigatinib (Pemazyre)

Pemigatinib is a small molecule kinase inhibitor that targets FGFR1, 2, and 3 by inhibiting FGFR1-3 phosphorylation and signaling. Indicated for previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test.

Infigratinib (Truseltiq)

Infigratinib is indicated for adults with previously treated, unresectable, locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement. 

Futibatinib (Lytgobi)

Indicated for previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma in adults harboring fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangements.

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IDH1 Inhibitors

Class Summary

Inhibitors of isocitrate dehydrogenase type 1 (IDH1) specifically inhibit a mutated form of IDH1 in the cytoplasm, which inhibits the formation of the oncometabolite, 2-hydroxyglutarate (2HG). This may lead to both an induction of cellular differentiation and an inhibition of cellular proliferation in IDH1-expressing tumor cells. IDH1, an enzyme in the citric acid cycle, is mutated in a variety of cancers; it initiates and drives cancer growth by both blocking cell differentiation and catalyzing the formation of 2HG. 

Ivosidenib (Tibsovo)

Indicated for locally advanced or metastatic cholangiocarcinoma in previously treated adults with IDH1 mutation.

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