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News
A new targeted therapy for bile duct cancer just got the FDA's stamp of approval. Lirafugratinib (Lyrfigtu) is now cleared for adults with previously treated, unresectable or metastatic cholangiocarcinoma harboring FGFR2 gene fusions or rearrangements. In the pivotal ReFocus trial, nearly half of patients responded to treatment, with strong survival outcomes and durable responses.
The FDA has approved lirafugratinib (Lyrfigtu, Elevar Therapeutics), an oral FGFR2 inhibitor, for adults with previously treated unresectable, locally advanced, or metastatic cholangiocarcinoma (bile duct cancer) carrying an FGFR2 gene fusion or rearrangement. About 8,000 new cases of this cancer are diagnosed in the U.S. each year, and treatment options have historically been limited.
What sets lirafugratinib apart from existing FGFR inhibitors (pemigatinib and futibatinib) is its irreversible, covalent-binding mechanism that selectively targets FGFR2 — minimizing off-target toxicities common with pan-FGFR inhibitors and overcoming resistance mutations that can emerge with earlier therapies. The recommended dose is 70 mg orally once daily.
By the Numbers:
Why it matters: This approval reinforces the critical role of molecular testing at diagnosis, so patients with FGFR2-positive cholangiocarcinoma can be matched to therapies most likely to benefit them. Elevar expects to launch the drug in the U.S. by Q4 2026.
