On September 23, 2026, the U.S. Food and Drug Administration (FDA) approved lirafugratinib (Lyrfigtu) for the treatment of adults with previously treated unresectable, locally advanced or metastatic cholangiocarcinoma harboring a fibroblast growth factor receptor 2 (FGFR2) gene fusion or other rearrangement. Lirafugratinib—a highly selective, oral small-molecule inhibitor of FGFR2—previously received Breakthrough Therapy and Orphan Drug designations.
Efficacy and Safety
Efficacy was evaluated in REFOCUS (ClinicalTrials.gov identifier NCT04526106), a multicenter, open-label, single-arm trial in 116 patients with unresectable or metastatic cholangiocarcinoma who were naive to FGFR inhibitor treatment and had received prior chemotherapy or chemoimmunotherapy. Patients received lirafugratinib at 70 mg once daily until disease progression or unacceptable toxicity.
The major efficacy outcome measures were objective response rate and duration of response as determined by an independent review committee according to RECIST v1.1. Objective response rate was 46% (95% confidence interval [CI] = 36–55) and median duration of response was 11.8 months (95% CI = 7.5–13.0).
The prescribing information includes warnings and precautions for ocular toxicity, hyperphosphatemia and soft tissue mineralization, and embryo-fetal toxicity.
Recommended Dosage
The recommended lirafugratinib dose is 70 mg orally once daily until disease progression or unacceptable toxicity.
