On September 30, 2022, the FDA accelerated the approval of futibatinib (Lytgobi; Taiho Oncology), a tyrosine kinase inhibitor, for the treatment of adults with previously treated unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma (CCA) and FGFR2 gene fusion or other rearrangements. Futibatinib received a breakthrough therapy designation for this indication.
This approval was based on results of the multicenter, open-label, single-arm TAS-120-101 clinical trial of 103 patients with unresectable, locally advanced, intrahepatic CCA associated with FGFR2 fusion or other rearrangements.
The primary end points were overall response rate and duration of response. All patients received 20 mg of futibatinib orally until disease progression or unacceptable adverse events. Of the 103 patients, 43 (42%) had a partial response (95% confidence interval [CI], 32-52) to futibatinib, with a median duration of response of 9.7 months (95% CI, 7.6-17.1).
The most common (≥20%) adverse events were nail problems, musculoskeletal pain, constipation, diarrhea, fatigue, dry mouth, alopecia, stomatitis, abdominal pain, dry skin, arthralgia, dysgeusia, dry eye, nausea, decreased appetite, urinary tract infection, palmar-plantar erythrodysesthesia syndrome, and vomiting.