FDA clears targeted cholangiocarcinoma therapy - Takeaways - MDSpire
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FDA clears targeted cholangiocarcinoma therapy

In the single-arm REFOCUS trial, 46% of patients responded to treatment, with a median response duration of 11.8 months.

  • By

  • Kathryn Wighton

  • September 24, 2026

  • 2 min

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  • 1

    The FDA approved lirafugratinib for adults with unresectable, locally advanced or metastatic cholangiocarcinoma with specific gene fusions.

  • 2

    Lirafugratinib is a kinase inhibitor administered at 70 mg once daily until disease progression or unacceptable toxicity.

  • 3

    The REFOCUS trial assessed lirafugratinib's efficacy, enrolling 116 patients previously treated with chemotherapy or chemoimmunotherapy.

  • 4

    In the trial, 46% of patients showed responses, with a median response duration of 11.8 months.

  • 5

    Safety warnings for lirafugratinib include ocular toxicity, hyperphosphatemia, soft tissue mineralization, and embryo-fetal toxicity.

Original Source(s)

FDA clears targeted cholangiocarcinoma therapy

  • by Kathryn Wighton

  • September 24, 2026

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