Multinational phase 3 trial of first-line pemigatinib for unresectable or metastatic holangiocarcinoma with FGFR2 rearrangement

Cholangiocarcinoma is associated with poor prognosis. The multinational phase 3 FIGHT-302 trial evaluated the FGFR2 inhibitor pemigatinib as first-line therapy for adult patients with advanced choliangiocarcinoma with FGFR2 rearrangement. Patients were randomized to receive daily pemigatinib (n=83) or gemcitabine-cisplatin chemotherapy (n=84). Primary endpoint was progression-free survival (PFS). Pemigatinib crossover was allowed for patients progressing on chemotherapy. The median PFS was 8.3 months in the pemigatinib group versus 6.8 months in the chemotherapy group (HR 0.58; p=0.0078); objective response rate was 47% versus 15%; median duration of response was 14.2 months versus 6.3 months. Median overall survival was similar (24.4 versus 25.0 months, respectively. In the crossover group (n=42) the median PFS on second-line pemigatinib was 8.1 months. Safety was consistent with the known profile of pemigatinib. The authors conclude that pemigatinib demonstrated prolonged median PFS compared with chemotherapy among patients with advanced FGFR2-rearranged cholangiocarcinoma.