Pemigatinib in Patients with Advanced Cholangiocarcinoma


by Dr. C.H. Weaver M.D. 11/2019

Results from the Phase 2 FIGHT-202 clinical trial evaluating the precision cancer medicine pemigatinib which selectively targets fibroblast growth factor receptor (FGFR) in advanced cholangiocarcinoma appear promising. Clinical trial results released at the European Society for Medical Oncology meeting will be used to support the planned submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for pemigatinib before the end of 2019.

About Cholangiocarcinoma

Cholangiocarcinoma is an uncommon cancer that forms in the cells lining the bile duct. It is classified based on its origin: intrahepatic cholangiocarcinoma (iCCA) occurs in the bile duct inside the liver and extrahepatic cholangiocarcinoma occurs in the bile duct outside the liver. Patients with cholangiocarcinoma are typically diagnosed at an advanced stage and their prognosis is poor. (2,3) FGFR2 fusions or rearrangements occur almost exclusively in iCCA, where they are observed in 10-16 percent of patients. (4-6)

About FGFR and Pemigatinib

Fibroblast growth factor receptors (FGFRs) play an important role in cancer cell growth and survival by enhancing migration and the formation of new blood vessels known as angiogenesis. A variety of genetic mutations in FGFRs are closely correlated with the development of various cancers. Pemigatininb binds to and inhibits FGFRs which prevents the growth and proliferation of FGFR overexpressing cancer cells.

The FIGHT (FIbroblast Growth factor receptor in oncology and Hematology Trials) clinical trial program includes ongoing phase 2 and 3 studies investigating safety and efficacy of pemigatinib therapy across several FGFR-driven malignancies.

In the fight Phase 2 clinical trial 107 patients harboring FGFR2 gene fusions or rearrangements were treated with pemigatinib and evaluated. Pemigatinib therapy was well tolerated and resulted in an overall response rate of 36% with median progression free survival of 6.9 months. Researchers reported 3 complete responses to treatment and the average survival duration for patients was reported to be 21 months.

These study results suggest that pemigatinib is a promising precision cancer medicine for patients with cholangiocarcinoma harboring FGFR2 gene fusions or rearrangements. Once FDA approved doctors will be able to better evaluate how best to use pemitatinib alone or in combination with other therapies to better improve the treatment outcomes for patients with cholangiocarcinoma and other cancers with FGFR2 genetic cancer driving mutations.


  1. Banales JM, et al. Nat Rev Gastroenterol Hepatol. 2016;13:261‒280.
  2. Uhlig J, et al. Ann Surg Oncol. 2019;26:1993–2000.
  3. Graham RP, et al. Hum Pathol. 2014;45:1630‒1638.
  4. Farshidfar F, et al. Cell Rep. 2017;18(11):2780–2794.
  5. Ross JS et al. The Oncologist. 2014;19:235–242.