FDA grants priority review to futibatinib for advanced cholangiocarcinoma
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The FDA granted priority review to futibatinib for treatment of certain patients with cholangiocarcinoma.
The designation applies to use of the agent by previously treated patients with locally advanced or metastatic disease who harbor FGFR2 gene rearrangements, including gene fusions.
Futibatinib (Taiho Pharmaceutical) is an oral selective and irreversible small-molecule inhibitor of FGFR1, 2, 3 and 4.
Approximately estimated 8,000 people in the United States are diagnosed each year with cholangiocarcinoma, a rare cancer of the bile ducts of the liver. An estimated 10% to 16% of those patients have tumors with FGFR2 gene rearrangements, including gene fusions.
“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,” Volker Wacheck, vice president for clinical development with Taiho Oncology, said in a company-issued press release.
A new drug application for this indication is based on results of the phase 2B FOENIX-CCA2 trial.
The trial included 103 patients with locally advanced or metastatic unresectable intrahepatic cholangiocarcinoma with FGFR2 gene rearrangements who had received at least one prior line of systemic therapy.
Study participants received 20 mg futibatinib once daily until disease progression or unacceptable toxicity. Objective response rate served as the primary endpoint and duration of response served as a key secondary endpoint.
Results showed an ORR per independent central review of 41.7%, and a median 9.7-month response duration. Nearly three-quarters (72%) of responses lasted at least 6 months.
Common treatment-related adverse events included hyperphosphatemia (85%), alopecia (33%) and dry mouth (30%). Two patients (1.9%) reported migraines.
The FDA previously granted breakthrough therapy designation to futibatinib for previously treated locally advanced or metastatic cholangiocarcinoma.
The agency is expected to make a decision on approval for this indication by Sept. 30.
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