The U.S. Food and Drug Administration has granted Priority Review to lirafugratinib, an oral FGFR2 inhibitor, for patients with pretreated cholangiocarcinoma carrying FGFR2 fusions or rearrangements. A regulatory decision is expected by September 27, 2026.
In the Phase 1/2 ReFocus trial, 114 FGFR inhibitor–naive patients showed a 46.5% objective response rate, including 2.6% complete responses, and a 96.5% disease control rate. Responses lasted a median of 11.8 months, with progression-free survival of 11.3 months and overall survival of 22.8 months.
The safety profile was manageable, with common side effects including nail toxicity (87.9%), palmar-plantar erythrodysesthesia (81.9%), and stomatitis (78.4%). Most adverse events were controlled with dose modifications, and only 4.3% of patients discontinued treatment. The trial focused on patients with unresectable or metastatic tumors who had progressed on chemotherapy but had not received prior FGFR inhibitors.