FDA Accepts Futibatinib NDA for Priority Review in Cholangiocarcinoma

A new drug application for futibatinib has been accepted for priority review in patients with previously treated locally advanced or metastatic cholangiocarcinoma with FGFR2 gene rearrangements.

A new drug application for futibatinib (TAS-120) has been accepted for priority review by the FDA for patients with previously treated locally advanced or metastatic cholangiocarcinoma who harbor a FGFR2 gene rearrangements, according to a press release from Taiho Oncology Inc.

This application is based on results from the phase 2b FOENIX-CCA2 trial (NCT02052778), which is assessing the therapy in patients with advanced solid tumors, including patients locally advanced or metastatic unresectable intrahepatic cholangiocarcinoma with FGFR2 gene rearrangements and who received 1 or more prior lines of therapy.

A prescription drug user fee act action date has been set for September 30, 2022.

“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 [cholangiocarcinoma], which has driven our pursuit with this investigational compound," Volker Wacheck, vice president of Clinical Development, Taiho Oncology, Inc, said in the press release.

The results from the study were previously presented at the 2021 American Association for Cancer Research Annual Meeting. Findings from the trial led to the FDA granting a breakthrough therapy designation for the treatment. The trial enrolled 103 patients who received 20 mg of futibatinib once daily until disease progression or unacceptable toxicity.

Patients had an overall response rate of 41.7% assessed by independent central review. The duration of response was 9.7 months, with 72% of patients experiencing a response lasting 6 months or more.

The most common treatment-related adverse effects among patients who received treatment were hyperphosphatemia (85%), alopecia (33%), and dry mouth (30%). Additionally, 1.9% of patients experienced migraine as a serious adverse effect.

Futibatinib is an investigation irreversible tyrosine kinase inhibitor of FGFR1, 2, 3, and 4, which is being studied for patients with solid tumors. It binds to the ATP binding pocket of FGFR 1 to 4 and inhibits the FGFR-mediated signal transduction pathways, in addition to reducing tumor cell proliferation and increased tumor cell death in those with FGFR1 to 4 genetic aberrations.

"This is a very important step towards our goal to deliver futibatinib to patients awaiting potential new treatment options. The Taiho group, working as one, will continue to do its utmost to deliver this agent to those in need,” Teruhiro Utsugi, senior managing director at Taiho Pharmaceutical, said in the press release.


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. March 30, 2022. Accessed March 30, 2022. https://prn.to/3u0Gvgd

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