The US Food and Drug Administration approved lirafugratinib (Lyrfigtu), a kinase inhibitor, for adults with previously treated unresectable, locally advanced or metastatic cholangiocarcinoma harboring a fibroblast growth factor receptor 2 gene fusion or other rearrangement, according to an FDA press release.

The FDA assessed the efficacy of lirafugratinib in the REFOCUS trial, which enrolled 116 patients with unresectable or metastatic cholangiocarcinoma previously treated with chemotherapy or chemoimmunotherapy. The multicenter, open-label, single-arm trial included patients who had not previously received a fibroblast growth factor receptor inhibitor.

Lirafugratinib was administered at 70 mg once daily and continued until disease progression or unacceptable toxicity. An independent review committee assessed objective response rate and duration of response, the trial’s major efficacy measures, using RECIST v1.1.

Responses were observed in 46% of patients, and the median response duration was 11.8 months.

Lirafugratinib is recommended at an oral dose of 70 mg once daily, with treatment continued until disease progression or unacceptable toxicity. Safety warnings and precautions address ocular toxicity, hyperphosphatemia and soft tissue mineralization, and embryo-fetal toxicity.

The FDA used the Real-Time Oncology Review pilot program and Assessment Aid during its evaluation of the application. The application was granted priority review, and lirafugratinib received breakthrough therapy and orphan drug designations.

Source: US Food and Drug Administration