The FDA approved Elevar Therapeutics’ lirafugratinib (Lyrfigtu) for adults with previously treated, unresectable, locally advanced or metastatic cholangiocarcinoma harboring FGFR2 fusions or other rearrangements. The decision is based on the multicenter, open-label REFOCUS trial (phase 1/2), which enrolled 116 patients who had not received an FGFR inhibitor and had received prior chemotherapy or chemoimmunotherapy. In REFOCUS, lirafugratinib achieved an objective response rate (ORR) of 46% and a median duration of response of 11.8 months, with additional data presented earlier at the 2026 ASCO Gastrointestinal Cancers Symposium. The FDA review also incorporated safety signals typical for FGFR inhibitors, including ocular toxicity and metabolic effects. For oncology, the approval expands targeted options in a genomically defined subset of a rare but aggressive cancer, where treatment choice is tightly linked to biomarker status. Clinicians will now weigh lirafugratinib against other systemic regimens in the post–first-line setting for FGFR2-driven disease.