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NewsThe FDA has approved lirafugratinib (Lyrfigtu) for adults with previously treated, unresectable or metastatic cholangiocarcinoma carrying an FGFR2 gene fusion or rearrangement. The decision was supported by the REFOCUS trial, where the drug achieved a 46% objective response rate and a median duration of response of nearly 12 months. It's a meaningful new option for patients with limited alternatives.
The FDA has approved lirafugratinib (Lyrfigtu), a targeted therapy for adults with previously treated, unresectable, locally advanced or metastatic cholangiocarcinoma — a rare and aggressive bile duct cancer — whose tumors harbor an FGFR2 gene fusion or rearrangement. This approval marks a new option for patients who have already gone through chemotherapy or chemoimmunotherapy and have not previously received an FGFR inhibitor.
The approval was backed by data from the REFOCUS trial, a multicenter, open-label, single-arm study of 116 patients. Participants received lirafugratinib at 70 mg orally once daily until disease progression or unacceptable toxicity.
By the Numbers:
Why it matters: Cholangiocarcinoma is notoriously difficult to treat, especially in advanced stages. For the subset of patients whose tumors carry FGFR2 alterations, lirafugratinib offers a molecularly targeted approach with durable responses — a significant step forward in precision oncology for this underserved patient population.
