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, under priority review, was based on results from the multicenter, open-label REFOCUS phase 1/2 trial. In REFOCUS, lirafugratinib achieved a 46% objective response rate, with a median duration of response of 11.8 months. Median progression-free survival was 11.3 months, and median overall survival was 22.8 months, according to the trial data discussed earlier at the 2026 ASCO Gastrointestinal Cancers Symposium. The prescribing information includes class-related safety warnings for ocular toxicity, hyperphosphatemia, and soft tissue mineralization, as well as embryo-fetal toxicity. Regulators positioned the therapy as a targeted option for FGFR2-altered bile duct cancers after progression on standard treatments.