FDA Accepts Futibatinib for Priority Review for FGFR2 Rearrangements and Fusions in Cholangiocarcinoma

The FDA has accepted Taiho Oncology’s investigational therapy futibatinib for priority review, the company announced Wednesday. The oral, selective small molecule inhibitor of FGFR1, 2, 3, and 4 would treat patients with FGFR2 gene rearrangements and gene fusions in patients with locally advanced or metastatic cholangiocarcinoma, also known as bile duct cancer.

According to the company’s statement, the FDA provided an anticipated Prescription Drug User Fee Act (PDUFA) date of September 30, 2022.

Cholangiocarcinoma, or CCA, is diagnosed in about 8000 people in the United States each year; while rare, its incidence is growing. Between 10% and 16% of those with cholangiocarcinoma have tumors with FGFR2 gene rearrangements.

Development of FGFR inhibitors ,which targetfibroblast growth factor receptors, is among the most important advances in the treatment of cholangiocarcinoma. Five-year survival rates for this type of cancer have historically been dismal, but in recent years there is tremendous innovation, given the number of treatment targets in these tumors.

“Given the lack of an accepted standard chemotherapy following the failure of first-line treatment, futibatinib could represent a significant opportunity for a targeted therapy in this subset of patients with CCA, which has driven our pursuit with this investigational compound,” said Volker Wacheck, vice president, Clinical Development, Taiho Oncology, Inc. “We look forward to working with the FDA as they consider the application for futibatinib under priority review.”

The new drug application is based on data from the pivotal phase 2b FOENIX-CCA2 trial, which involved 103 patients with locally advanced or metastatic unresectable intrahepatic cholangiocarcinoma harboring FGFR2 gene rearrangements including fusions who had received one or more prior lines of systemic therapy. Patients received futibatinib 20 mg once daily until disease progression or unacceptable toxicity.

The primary endpoint was an objective response rate of 41.7%. The key secondary endpoint of duration of response showed a median of 9.7 months (72% of responses ≥6 months). The most common treatment-related adverse events in the trial were hyperphosphatemia (85%), alopecia (33%) and dry mouth (30%). The only serious adverse reaction reported in more than 1 patient enrolled in the FOENIX-CCA2 trial was migraine (1.9%).