FDA Approves Lyrfigtu for Previously Treated FGFR2-Positive Cholangiocarcinoma

The Food and Drug Administration (FDA) approved Lyrfigtu (lirafugratinib) for adults with previously treated, unresectable, locally advanced or metastatic cholangiocarcinoma with an FGFR2 gene fusion or other rearrangement.

Cholangiocarcinoma is a cancer that begins in the bile ducts. Unresectable means the cancer cannot be removed with surgery, while metastatic cancer has spread to other parts of the body.

The approval was based on findings from the REFOCUS trial, which included 116 patients who had previously received chemotherapy or chemoimmunotherapy and had not previously received an FGFR inhibitor.

How effective was Lyrfigtu for FGFR2-positive cholangiocarcinoma?

In the REFOCUS trial, Lyrfigtu led to an objective response rate of 46%. An objective response means the cancer shrank by a predetermined amount on scans.

Among patients whose cancer responded to treatment, the median duration of response was 11.8 months. Duration of response measures how long the cancer continued to respond after treatment began working.

Both objective response rate and duration of response were major measures of how well Lyrfigtu worked in the trial.

Who was included in the REFOCUS cholangiocarcinoma trial?

REFOCUS was a multicenter, open-label, single-arm trial that included 116 patients with unresectable or metastatic cholangiocarcinoma.

All patients had previously received chemotherapy or chemoimmunotherapy and had not been treated with an FGFR inhibitor.

Patients received 70 milligrams of Lyrfigtu by mouth once daily. Treatment continued until the cancer worsened or side effects became unacceptable.

An independent review committee evaluated patients' responses to treatment using standard criteria for measuring changes in tumor size on scans.

What side effects should patients know about with Lyrfigtu?

The prescribing information for Lyrfigtu includes warnings and precautions for eye-related side effects, high phosphate levels in the blood, mineral buildup in soft tissues and potential harm to an unborn baby.

The FDA recommends a dose of 70 milligrams of Lyrfigtu taken by mouth once daily until the cancer progresses or side effects become unacceptable.

The FDA granted the application priority review. Lyrfigtu also received breakthrough therapy and orphan drug designations.

Reference

1. “FDA approves lirafugratinib for previously treated, unresectable, locally advanced or metastatic cholangiocarcinoma.” U.S. Food and Drug Administration. Sept. 23, 2026.

For more news on cancer updates, research and education, don't forget to subscribe to CURE®'s newsletters here.