Pemigatinib is a medication used to treat previously treated, unresectable locally advanced or metastatic bile duct cancer with FGFR2 or other rearrangements. This study aims to examine the antitumor activity and safety of pemigatinib in patients with previously treated locally advanced or metastatic cholangiocarcinoma with or without FGFR2 or other rearrangements.
This open-label, single-arm, multicohort, multicenter, phase 2 study included a total of 146 patients aged 18 years or older with locally advanced or metastatic cholangiocarcinoma. The patients were divided into three cohorts: cohort A with patients with FGFR2 fusions or rearrangements, cohort B with patients with other FGF/FGFR alterations, and cohort C with patients with no FGF/FGFR alterations. All the patients were assigned to a starting dose of pemigatinib. The primary outcome of the study was an objective response among FGFR2 fusions and other rearrangements.
During the median follow-up of 17.8 months, 35.5% of patients with FGFR2 fusions or rearrangements achieved an objective response. Hyperphosphataemia was the only commonly occurring all-grade adverse event. A total of 64% of the patients had grade 3 or worse adverse events, and 45% had serious adverse events.
The research concluded that pemigatinib could be an effective treatment in patients with previously treated locally advanced or metastatic cholangiocarcinoma with FGFR2 or other rearrangements.