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News|Articles|September 23, 2026

FDA Approves Lirafugratinib for Pretreated, Locally Advanced or Metastatic Cholangiocarcinoma

Author(s)OncLive Staff
Fact checked by: Ashling Wahner
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Key Takeaways

  • REFOCUS used a nonrandomized, single-arm, multicenter, open-label design intended to estimate antitumor activity in a molecularly defined cholangiocarcinoma population.
  • Eligibility required unresectable or metastatic cholangiocarcinoma with FGFR2 fusion/rearrangement after prior chemotherapy or chemoimmunotherapy, with no prior FGFR inhibitor exposure.
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The FDA has approved lirafugratinib (Lyrfigtu) for the treatment of adult patients with previously treated unresectable, locally advanced or metastatic cholangiocarcinoma harboring an FGFR2 gene fusion or other rearrangement.

The FDA has approved lirafugratinib (Lyrfigtu) for the treatment of adult patients with previously treated unresectable, locally advanced or metastatic cholangiocarcinoma harboring an FGFR2 gene fusion or other rearrangement.1

This regulatory decision was backed by data from the REFOCUS trial, (NCT04526106), in which lirafugratinib elicited an ORR of 46% (95% CI, 36%, 55%) and a median DOR of 11.8 months (95% CI, 7.5, 13.0).

What was the design of the REFOCUS trial?

This multicenter, open-label, single-arm trial enrolled 116 patients with unresectable or metastatic cholangiocarcinoma who had not previously received an FGFR inhibitor and had been previously treated with chemotherapy or chemoimmunotherapy. Patients were treated with lirafugratinib at 70 mg once daily until disease progression or unacceptable toxicity.

References

FDA approves lirafugratinib for previously treated, unresectable, locally advanced or metastatic cholangiocarcinoma. FDA. September 23, 2026. Accessed September 23, 2026. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-lirafugratinib-previously-treated-unresectable-locally-advanced-or-metastatic

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