Key takeaways:

• FDA approved Lyrfigtu for previously treated FGFR2-mutated unresectable, locally advanced or metastatic cholangiocarcinoma.
• REFOCUS trial data showed a 46% ORR and an 11.8-month median duration of response.

The FDA approved lirafugratinib — a kinase inhibitor — for adults with previously treated unresectable, locally advanced or metastatic cholangiocarcinoma.

The indication for lirafugratinib (Lyrfigtu, Elevar Therapeutics) applies to patients with a fibroblast growth factor receptor 2 (FGFR2) gene fusion or other rearrangement.

The FDA approved lirafugratinib — a kinase inhibitor — for adults with previously treated unresectable, locally advanced or metastatic cholangiocarcinoma.

The FDA based its decision on results from the phase 1/2 REFOCUS trial, which enrolled 116 patients with unresectable or metastatic cholangiocarcinoma who had received prior chemotherapy or chemoimmunotherapy but had not previously received an FGFR inhibitor.

Patients received 70 mg lirafugratinib orally once daily until disease progression or unacceptable toxicity.

Results showed an ORR of 46% (95% CI, 36%-55%), with a median duration of response of 11.8 months (95% CI, 7.5-13).

Prescribing information for lirafugratinib includes warnings and precautions for ocular toxicity, hyperphosphatemia and soft tissue mineralization, and embryo-fetal toxicity.

The recommended dose is 70 mg orally taken once daily until disease progression or unacceptable toxicity.

The FDA granted lirafugratinib priority review, adding to its earlier breakthrough therapy and orphan drug designations.