for cholangiocarcinoma
FDA approved Lyrfigtu (lirafugratinib) on September 23, 2026 — four days ahead of the September 27 PDUFA target — for adults with previously treated unresectable, locally advanced or metastatic cholangiocarcinoma harboring an FGFR2 fusion or other rearrangement. Approval based on the Phase 1/2 REFOCUS trial (n=116). Selective FGFR2 inhibitor differentiated from pan-FGFR inhibitors.
Cholangiocarcinoma, also called bile duct cancer, forms in the ducts that carry bile, causing abdominal pain, yellowing skin, weight loss, itching, and fever. Lirafugratinib is an oral drug designed to block FGFR2, a growth signal that, when fused or rearranged, drives tumor cells to multiply. Because that abnormal signal appears to fuel this cancer, blocking it could potentially slow tumor growth in people whose tumors carry this change.
The goal of this clinical trial is to evaluate if lirafugratinib is efficacious and safe to treat adult patients with previously treated, unresectable, locally advanced or metastatic solid tumors (excluding cholangiocarcinoma) harboring FGFR2 fusion or rearrangement. Participants will: * Take lirafugratinib regularly as instructed by their study doctor
Source: ClinicalTrials.gov