FDA approves lirafugratinib for previously treated bile duct cancer

The FDA approved lirafugratinib (Lyrfigtu) for adults with previously treated, unresectable, locally advanced or metastatic cholangiocarcinoma, also called bile duct cancer, that has an FGFR2 gene fusion or other rearrangement. The FDA announced the approval on September 23, 2026.

The FDA said efficacy was evaluated in REFOCUS, a trial of 116 patients who had received prior chemotherapy or chemoimmunotherapy and had not previously received an FGFR inhibitor. The objective response rate was 46% (95% CI: 36, 55), and median duration of response was 11.8 months (95% CI: 7.5, 13.0), according to the FDA.

The FDA prescribing information includes warnings and precautions for ocular toxicity, hyperphosphatemia and soft tissue mineralization, and embryo-fetal toxicity. MedPage Today reported that Elevar Therapeutics said the drug will be available in the U.S. before the end of the year.