On April 27, 2023 Taiho Pharmaceutical Co., Ltd. and its European subsidiary Taiho Oncology Europe GmbH reported that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending the conditional marketing authorization (CMA) of futibatinib for the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma(CCA) with a fibroblast growth factor receptor 2(FGFR2) fusion or rearrangement that have progressed after at least one prior line of systemic therapy (Press release, Taiho, APR 27, 2023, View Source [SID1234630624]).

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The CHMP’s opinion to recommend the use of futibatinib is now being reviewed by the full EMA. Final marketing authorizations for medical products in the European Union (EU) rests with the European Commission. Each year, approximately 6,000-8,000 individuals in Europe are diagnosed with CCA,1 a rare cancer of the bile ducts of the liver, and approximately 0.3-6 people per 100,000 individuals live with CCA worldwide.

"CCA is an aggressive cancer, and the current five-year survival rate is very poor, which underscores the need for new treatment options" said Peter Foertig, MD, Vice President, Medical Affairs, Taiho Oncology Europe. "We look forward to working with the European Medicines Agency as it reviews the application for marketing authorization."

The positive CHMP opinion on futibatinib is based on data from the pivotal Phase 2 FOENIX-CCA2 ※ trial in 103 patients with locally advanced or metastatic unresectable intrahepatic (inside the liver) CCA, harboring FGFR2 fusions or rearrangements who had received one or more prior lines of systemic therapy. Patients in the trial received futibatinib 20mg once daily until disease progression
or unacceptable toxicity. The trial’s primary endpoint was an objective response
rate (ORR).

※ FOENIX-CCA2：PHASE 1/2 STUDY OF TAS-120 IN PATIENTS WITH ADVANCED SOLID TUMORS Harboring FGF/FGFR Aberrations; FGFR Oral SElective Novel Inhibitor X [across] tumors

Results from the trial were published in the January 19, 2023, issue of The New England Journal of Medicine.

"As someone who treats patients with CCA, I am encouraged by these data and the positive news from CHMP," said Professor Arndt Vogel, MD, Senior Consultant, Department of Gastroenterology, Hepatology and Endocrinology, Hannover Medical School, Germany. "The study findings underscore the importance of molecular profiling for patients with CCA and other diseases whenever possible to potentially help improve outcomes. Indeed, the ESMO (Free ESMO Whitepaper) Clinical Practice Guidelines recommend FGFR inhibitors for the treatment of patients with CCA and FGFR2 fusions who have progressed after one or more prior lines of systemic therapy."

Takeshi Sagara Ph.D., Managing Director, Clinical Development and Medical Affairs, Discovery and Preclinical Research at Taiho Pharmaceutical, added "The global Taiho group will continue its efforts to bring futibatinib as a new treatment option to patients with CCA around the world, and the positive CHMP opinion represents a big step for Taiho in Europe to deliver innovative drugs to help patients with CCA in their journey".

About Futibatinib
Futibatinib (TAS-120) is an oral, potent, selective, and irreversible tyrosine kinase 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 covalently binds to the ATP binding pocket of FGFR1-4
resulting in the inhibition of FGFR-mediated signal transduction pathways, reduced tumor cell proliferation in tumors with FGFR1-4 genetic aberrations.

In Japan, a new drug application for futibatinib has been submitted to the Japanese Ministry of Health, Labour and Welfare, as a treatment for previously treated locally advanced or metastatic biliary tract cancer harboring FGFR2 gene rearrangements, including gene fusions, on July 2022. Furthermore, in September 2022, the U.S. Food and Drug Administration (FDA) approved futibatinib for the treatment of adult patients with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma (iCCA) harboring fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangements.