The Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application (NDA) for futibatinib in the treatment of patients with previously treated locally advanced or metastatic cholangiocarcinoma harboring FGFR2gene rearrangements, including gene fusions. 

Futibatinib is an investigational, oral, potent, selective and irreversible small molecular inhibitor of FGFR1, 2, 3, and 4. The NDA submission is supported by data from the ongoing pivotal, open-label phase 2b FOENIX-CCA2 trial ( Identifier: NCT02052778) evaluating futibatinib in 103 adults with locally advanced or metastatic cholangiocarcinoma harboring FGFR2 gene rearrangements, including gene fusions who received 1 or more prior lines of systemic therapy. Patients received futibatinib 20mg orally once daily until disease progression or unacceptable toxicity. 

Results showed that treatment with futibatinib achieved an objective response rate of 41.7% (primary endpoint) as assessed by independent central review. The median duration of response (secondary endpoint) was 9.7 months, with 72% of responses lasting at least 6 months.

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As for safety, the common treatment-related adverse events were hyperphosphatemia (85%), alopecia (33%), and dry mouth (30%). A serious adverse reaction reported in more than 1 patient was migraine (1.9%).

A Prescription Drug User Fee Act (PDUFA) target date of September 30, 2022 has been set for this application.

“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.”

Based on trial data, in 2021 the FDA granted Breakthrough Therapy designation to futibatinib for the treatment of patients with previously treated locally advanced or metastatic cholangiocarcinoma.


U.S. Food and Drug Administration (FDA) accepts for Priority Review Taiho Oncology’s New Drug Application for futibatinib for cholangiocarcinoma. News release. Taiho Oncology, Inc. Accessed March 30, 2022.

This article originally appeared on MPR