The FDA approved Elevar Therapeutics’ Lyrfigtu (lirafugratinib) for adults with previously treated unresectable, locally advanced or metastatic cholangiocarcinoma harboring FGFR2 fusions or other rearrangements. The decision was supported by data from the multicenter, open-label REFOCUS phase 1/2 trial. In REFOCUS, the FDA relied on objective response rate and duration of response assessed using RECIST v1.1. The trial included 116 patients who had not received an FGFR inhibitor and had received prior chemotherapy or chemoimmunotherapy, receiving lirafugratinib 70 mg once daily until progression or unacceptable toxicity. Efficacy results reported include a 46% objective response rate and a median duration of response of 11.8 months, alongside additional context from prior ASCO GI Cancers Symposium presentations. The approval label includes safety warnings typical for FGFR inhibitors, including ocular toxicity and risks related to embryo-fetal effects and hyperphosphatemia.