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

MDSpire News

Objective:

To report the FDA approval of lirafugratinib for adults with previously treated unresectable, locally advanced or metastatic cholangiocarcinoma.

Approach:

Trial Design: The REFOCUS trial was a multicenter, open-label, single-arm study that enrolled 116 patients with unresectable or metastatic cholangiocarcinoma previously treated with chemotherapy or chemoimmunotherapy.
Treatment Administration: Lirafugratinib was administered at 70 mg once daily until disease progression or unacceptable toxicity.
Efficacy Assessment: An independent review committee assessed the objective response rate and duration of response using RECIST v1.1.

Key Findings:

Responses were observed in 46% of patients.
The median response duration was 11.8 months.

Limitations:

The trial included patients who had not previously received a fibroblast growth factor receptor inhibitor.

Conclusion:

Lirafugratinib received breakthrough therapy and orphan drug designations, with safety warnings for ocular toxicity, hyperphosphatemia, and embryo-fetal toxicity.