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The FGFR inhibitor pemigatinib shrank tumours in more than a third of patients with previously treated intrahepatic cholangiocarcinoma carrying FGFR2 fusions, the first targeted drug approved for the disease.
Phase 2 study of 146 patients with previously treated locally advanced or metastatic cholangiocarcinoma, including 107 with FGFR2 fusions or rearrangements, treated with pemigatinib 13.5 mg daily on a two-weeks-on, one-week-off schedule.
In FGFR2-rearranged patients objective response was 35.5 percent with median duration of response 7.5 months and median progression-free survival 6.9 months; no responses were seen in patients with other FGF/FGFR alterations. Hyperphosphataemia was almost universal.
FGFR2 fusion testing is standard in intrahepatic cholangiocarcinoma, and pemigatinib (with futibatinib) is the second-line targeted option for fusion-positive disease.
Shares Intrahepatic cholangiocarcinoma, The Lancet Oncology.
Shares FIGHT-202, Pemigatinib, Intrahepatic cholangiocarcinoma.