Targeted Therapy for Bile Duct Cancer

As researchers learn more about the changes in cells that cause cancer, they've developed drugs to target some of these changes. These targeted drugs work differently from standard chemotherapy (chemo) drugs. They sometimes work when standard chemo drugs don’t, and they often have different side effects.

FGFR2 inhibitor

FGFRs (fibroblast growth factor receptors) are proteins on cells that help them grow and divide normally. A small number (about 15%) of people with bile duct cancer within the liver have changes in the genes that make FGFRs, which result in abnormal FGFR proteins that cause cells to grow out of control and turn into cancer.

Pemigatinib (Pemazyre), a FGFR2 inhibitor, blocks the abnormal FGFR2 protein in bile duct cancer cells and keeps them from growing and spreading to other places. It can be used to treat some advanced bile duct cancers that cannot be removed by surgery or have spread to distant areas after at least one previous chemotherapy treatment. For this drug to work, your cancer must have the abnormal FGFR2 gene, so your cancer will be tested before starting the drug.

This drug is given as a tablet by mouth once a day for 2 weeks, followed by one week off, then started again.

Possible side effects of a FGFR2 inhibitor

The most common side effects of pemigatinib are having too much or too little phosphate in the blood, hair loss, diarrhea, constipation, nail problems, fatigue, taste changes, nausea, vomiting, dry mouth or mouth sores, loss of appetite, dry skin, dry eye or other eye problems, and joint, abdominal, or back pain.

More information about targeted therapy

To learn more about how targeted drugs are used to treat cancer, see Targeted Therapy.

To learn about some of the side effects listed here and how to manage them, see Managing Cancer-related Side Effects.

The American Cancer Society medical and editorial content team

Our team is made up of doctors and oncology certified nurses with deep knowledge of cancer care as well as journalists, editors, and translators with extensive experience in medical writing.

Abou-Alfa GK, Sahai V, Hollebecque A, Vaccaro G, Melisi D, Al-Rajabi R, et al. Pemigatinib for previously treated, locally advanced or metastatic cholangiocarcinoma: a multicentre, open-label, phase 2 study. Lancet Oncol. 2020 May;21(5):671-684. doi: 10.1016/S1470-2045(20)30109-1. Epub 2020 Mar 20.

DeLeon TT, Ahn DH, Bogenberger JM, et al. Novel targeted therapy strategies for biliary tract cancers and hepatocellular carcinoma. Future Oncol. 2018;14(6):553-566.

Hoyos S, Navas M-C, Restrepo J-C, Botero RC. Current controversies in cholangiosarcoma. BBA - Molecular Basis of Dis. 2108;1864:1461-1467.

Lombardi P, Marino D, Fenocchio E, et al. Emerging molecular target antagonists for the treatment of biliary tract cancer. Expert Opinion on Emerging Drugs. 2018;23(1):63-75.

National Comprehensive Cancer Network, Clinical Practice Guidelines in Oncology (NCCN Guidelines®), Hepatobiliary Cancers, Version 2.2018 -- June 7, 2018. Accessed at www.nccn.org/professionals/physician_gls/pdf/hepatobiliary.pdf on June 26, 2018.

Rizvi S, Khan SA, Hallemeier CL, Kelley RK, Gores GJ. Cholangiocarcinoma: Evolving concepts and therapeutic strategies. Nat Rev Clin Oncol. 2018;15(2):95-111.

Valle JW, Lamarca A, Goyal L, Barriuso J, Zhu AX. New Horizons for Precision Medicine in Biliary Tract Cancers. Cancer Discov. 2017;7(9):943-962.  

References

Abou-Alfa GK, Sahai V, Hollebecque A, Vaccaro G, Melisi D, Al-Rajabi R, et al. Pemigatinib for previously treated, locally advanced or metastatic cholangiocarcinoma: a multicentre, open-label, phase 2 study. Lancet Oncol. 2020 May;21(5):671-684. doi: 10.1016/S1470-2045(20)30109-1. Epub 2020 Mar 20.

DeLeon TT, Ahn DH, Bogenberger JM, et al. Novel targeted therapy strategies for biliary tract cancers and hepatocellular carcinoma. Future Oncol. 2018;14(6):553-566.

Hoyos S, Navas M-C, Restrepo J-C, Botero RC. Current controversies in cholangiosarcoma. BBA - Molecular Basis of Dis. 2108;1864:1461-1467.

Lombardi P, Marino D, Fenocchio E, et al. Emerging molecular target antagonists for the treatment of biliary tract cancer. Expert Opinion on Emerging Drugs. 2018;23(1):63-75.

National Comprehensive Cancer Network, Clinical Practice Guidelines in Oncology (NCCN Guidelines®), Hepatobiliary Cancers, Version 2.2018 -- June 7, 2018. Accessed at www.nccn.org/professionals/physician_gls/pdf/hepatobiliary.pdf on June 26, 2018.

Rizvi S, Khan SA, Hallemeier CL, Kelley RK, Gores GJ. Cholangiocarcinoma: Evolving concepts and therapeutic strategies. Nat Rev Clin Oncol. 2018;15(2):95-111.

Valle JW, Lamarca A, Goyal L, Barriuso J, Zhu AX. New Horizons for Precision Medicine in Biliary Tract Cancers. Cancer Discov. 2017;7(9):943-962.  

Last Revised: May 8, 2020

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