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NewsThe FDA has approved lirafugratinib (Lyrfigtu) for adults with previously treated, unresectable or metastatic cholangiocarcinoma harboring an FGFR2 gene fusion or rearrangement. Data from the phase 1/2 REFOCUS trial showed a 46% overall response rate and a nearly 12-month median duration of response. The drug is taken orally once daily and carries warnings for ocular toxicity and hyperphosphatemia.
The FDA has approved lirafugratinib (Lyrfigtu, Elevar Therapeutics), a kinase inhibitor, for adults with previously treated unresectable, locally advanced, or metastatic cholangiocarcinoma — a rare and aggressive bile duct cancer — whose tumors carry a fibroblast growth factor receptor 2 (FGFR2) gene fusion or other rearrangement. The approval adds a new targeted option for patients who have already received chemotherapy or chemoimmunotherapy but have not yet been treated with an FGFR inhibitor.
The FDA's decision was based on results from the phase 1/2 REFOCUS trial, which enrolled 116 patients. Lirafugratinib is taken orally at 70 mg once daily until disease progression or unacceptable toxicity. The drug previously received breakthrough therapy, orphan drug, and priority review designations — reflecting the urgency of the unmet need in this population.
By the Numbers:
Why it matters: Cholangiocarcinoma is notoriously difficult to treat, and options after first-line therapy are limited. This approval gives oncologists a targeted, biomarker-driven tool for a subset of patients with FGFR2-altered tumors, potentially extending meaningful responses in a disease with poor prognosis.
