Durable response with pemigatinib in cholangiocarcinoma

The selective, potent, oral inhibitor of fibroblast growth factor receptor1-3 (FGFR1-3) pemigatinib induces a durable response in patients with previously treated advanced or metastatic cholangiocarcinoma, as shown by the open label study FIGHT-202, whose results have been published recently on ESMO Open.

Authors enrolled 147 with previously treated advanced or metastatic cholangiocarcinoma with or without FGF/FGFR alterations; participants received once-daily oral pemigatinib 13.5 mg in 21-day cycles (2 weeks on, 1 week off) until disease progression or unacceptable toxicity. The results from the extended follow-up period (median 45.4 months) show that objective response rate in the group with FGFR2 fusions or rearrangements was 37%, with complete and partial responses observed in 3 and 37 patients, respectively. Median duration of response was 9.1 months and median progression-free survival and overall survival were 7 and 17.5 months, respectively. 10.2% of patients experienced treatment-related adverse events leading to treatment discontinuation. As authors conclude, «This final analysis of FIGHT-202 demonstrated continued durable response, prolonged overall survival and manageable adverse events in patients with previously treated advanced or metastatic cholangiocarcinoma with FGFR2 fusions or rearrangements, further supporting regulatory approvals of pemigatinib based on this single-arm, phase II study. These results highlight the need for early molecular testing in cholangiocarcinoma. The phase III FIGHT-302 study will further elucidate the role of FGFR inhibitors in biomarker-selected cholangiocarcinoma. Routine comprehensive genomic profiling is needed to discover novel actionable FGFR2 alterations and identify patients who might benefit from FGFR inhibition».