The U.S. Food and Drug Administration (FDA) has approved the kinase inhibitor, Pemazyre (pemigatinib), to treat adults with 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.
Cholangiocarcinoma is a rare cancer that forms in the bile duct. Patients with cholangiocarcinoma are often diagnosed at a later stage when the prognosis is poor.
The disease can be classified based on whether the cancer is in the bile duct within the liver [intrahepatic cholangiocarcinoma (iCCA)] or outside the liver (extrahepatic cholangiocarcinoma). FGFR2 fusions or rearrangements occur in about 10-16% of patients with iCCA. FGFRs are believed to play an important role in tumor cell proliferation and survival, migration and angiogenesis.
This is first FDA-approved treatment for this indication.
The accelerated approval was largely based on data from a Phase 2 open-label study (FIGHT-202 study). Continued approval may be contingent on verification and description of clinical benefit in a confirmatory trial(s) that are currently underway.
The FIGHT-202 study was a multi-center, open-label, single-arm study that evaluated pemigatinib safety/efficacy in adults with cholangiocarcinoma. In patients harboring FGFR2 fusions or rearrangements (Cohort A), pemigatinib monotherapy resulted in an overall response rate of 36%, and median duration of response of 9.1 months.
Warnings and precautions for pemigatinib include dry or inflamed eyes, inflamed cornea, increased tears and a disorder of the retina; high levels of phosphate in the blood; and, for women who are pregnant, a risk of harm to the unborn baby or loss of pregnancy.
In a news release, Ghassan Abou-Alfa, M.D., Memorial Sloan Kettering Cancer Center said, “It is encouraging to have a new targeted treatment option for patients who historically have had limited options after first-line chemotherapy or surgery, in which relapse rates remain high.”
The FDA is also expected to approve FoundationOne®CDx as the companion diagnostic for pemigatinib to help identify patients with FGFR2 fusions or rearrangements.
