RARE Daily

FDA Grants Breakthrough Therapy to Taiho’s Treatment for Advanced Cholangiocarcinoma

April 7, 2021

Rare Daily Staff

The U.S. Food and Drug Administration granted Breakthrough Therapy designation to Taiho Oncology’s futibatinib for the treatment of patients with previously treated locally advanced or metastatic cholangiocarcinoma harboring FGFR2 gene rearrangements, including gene fusions.

Cholangiocarcinoma (CCA), also known as bile duct cancer, is rare with about 8,000 people in the U.S. diagnosed each year. Although CCA can occur at younger ages, the average age of people in the U.S. diagnosed with CCA is 71, and the five-year survival rate of intrahepatic CCA is just 9 percent. Currently, standard treatment options are limited to radiation, palliative therapy, liver transplantation, surgery, chemotherapy and interventional radiology.

“Patients living with locally advanced and metastatic cholangiocarcinoma, or bile duct cancer, currently have a poor prognosis, particularly since there is no standard treatment after the failure of first-line chemotherapy,” said Martin Birkhofer, senior vice president and chief medical officer of Taiho Oncology, a subsidiary of Japanese pharmaceutical Otsuka. “We are pleased that the FDA has recognized the potential benefit of futibatinib in previously treated CCA patients. We look forward to continued dialogue with FDA and other health authorities as we work toward global availability of futibatinib for cholangiocarcinoma patients.”

Futibatinib (TAS-120) is an experimental, oral, potent, selective, and irreversible small-molecule inhibitor of FGFR1, 2, 3 and 4 being studied as a potential treatment for patients with advanced solid tumors with FGFR1-4 genetic aberrations, including cholangiocarcinoma, who were previously treated with chemotherapy or other therapies. Futibatinib selectively and irreversibly binds to the ATP binding pocket of FGFR1-4 resulting in the inhibition of FGFR-mediated signal transduction pathways, reduced tumor cell proliferation and increased tumor cell death in tumors with FGFR1-4 genetic aberrations.

The FDA granted this designation based on efficacy and safety results from the phase 2 FOENIX-CCA2 study, which will be presented at the American Association for Cancer Research (AACR) Annual Meeting 2021, taking place April 9 to 14, 2021.

“We are very pleased with the designation of futibatinib as a breakthrough therapy by the FDA,” said Teruhiro Utsugi, senior managing director at Taiho. “We will continue to advance our research and development efforts to deliver futibatinib, discovered in our research center, as one of the agents which may benefit cholangiocarcinoma patients around the world awaiting new treatment options.”

Breakthrough Therapy designation is intended to expedite the development and review of drugs for serious or life-threatening conditions. The criteria for BTD require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy. In May 2018, the FDA granted futibatinib orphan drug status for the treatment of cholangiocarcinoma.

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