The FDA has approved 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 phase 1/2 trial (n=116) evaluating objective response rate and duration of response per RECIST v1.1. In REFOCUS, lirafugratinib generated a 46% objective response rate with a median duration of response of 11.8 months. Median progression-free survival was reported as 11.3 months, with nearly half of patients progression-free at one year, and median overall survival of 22.8 months. The prescribing information includes ocular toxicity, hyperphosphatemia and soft-tissue mineralization, plus embryo-fetal toxicity warnings. Clinically, the approval fills a treatment gap for a rare, aggressive cancer subtype that often progresses after prior chemotherapy or chemoimmunotherapy. For developers, it also reinforces how molecular stratification is continuing to shape regulatory pathways in solid tumors beyond breast and lung cancer. Regulators granted the approval under priority review, positioning Lyrfigtu as an on-target oral option for the subset of patients with FGFR2-driven disease.