News Releases

Taiho Pharmaceutical Co., Ltd. (hereinafter “Taiho”) announced today that it has submitted to the Japanese Ministry of Health, Labour and Welfare a new drug application for futibatinib (TAS-120), a FGFR inhibitor as a treatment for previously treated locally advanced or metastatic biliary tract cancer harboring FGFR2 gene rearrangements, including gene fusions.

The NDA submitted is based on the data from the Phase 2 FOENIX-CCA2 trial. FOENIX-CCA2 trial was a Phase 2 trial in 103 patients with locally advanced or metastatic unresectable intrahepatic cholangiocarcinoma harboring FGFR2 gene rearrangements including gene fusions. In the trial, patients who had received one or more prior lines of systemic therapy received futibatinib 20 mg once daily until disease progression or unacceptable toxicity. The trial’s primary endpoint was objective response rate (ORR) ^※.

※ORR(objective response rate): Percentage of patients with objective evidence of response to anticancer treatment and other treatments

Furthermore, a companion diagnostic device to detect FGFR2 gene rearrangements including gene fusions is being jointly developed in Japan with Sysmex Corporation.

Taiho will hereafter continue its effort to secure the approval of futibatinib, aiming to deliver this new treatment to patients in needs.

Biliary tract cancer is a general term for cancer that develops in the biliary tract and is classified into bile duct cancer, gall bladder cancer, and papillary cancer, depending on the site of origin. Intrahepatic cholangiocarcinoma, which occurs in the bile ducts within the liver, is included as biliary tract cancer. According to the National Cancer Center, the annual incidence of biliary tract cancer, including intrahepatic and extrahepatic cholangiocarcinoma, gallbladder cancer, and papillary cancer, is approximately 25,000 in Japan.¹ Currently, there are few treatment options, and no standard treatment for locally advanced or metastatic biliary tract cancer that has progressed after 1^st line treatment.

Futibatinib (TAS-120) is an investigational, 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 biliary tract cancer, 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. For details of the FOENIX-CCA2 trial, please refer to ClinicalTrials.gov (https://clinicaltrials.gov/ct2/home trial ID：NCT02052778).

FOENIX-CCA2 trial：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

Overseas, in May 2018 futibatinib has been granted orphan drug status for the treatment of cholangiocarcinoma, and also have received Breakthrough Designation for the treatment of patients with previously treated locally advanced or metastatic cholangiocarcinoma harboring FGFR2 gene rearrangements, including gene fusions in April 2021 from the US FDA. March 2022, FDA accepted the New Drug Application (NDA) for futibatinib under priority review.

FGFRs belong to a family of receptor-type tyrosine kinases involved in angiogenesis, wound healing and embryonic development. Fibroblast growth factors (FGFs) and their receptors (FGFR1-4) are expressed on diverse cell types and regulate cell growth, survival, migration and differentiation. Recently, FGF/FGFR gene abnormalities have been reported in several types of cancer, and have attracted attention as candidate driver genes for cancer.

In line with its mission of “shaping the advancement of healthcare,” which is defined in the “Sysmex Way,” the corporate philosophy of the Sysmex Group, Sysmex works to contribute to the development of healthcare and the healthy lives of people. Sysmex conducts integrated R&D, manufacturing and sales, and provides support services for its instruments, reagents and software for in vitro testing of blood, urine and other bodily fluids. Sysmex supplies its products to medical institutions in more than 190 countries and regions throughout the world. In recent years, Sysmex has been expanding its business in the life science domain, using proprietary technologies to create new testing and diagnostic value, realize healthcare that is optimized for individual patients, and help reduce patients’ burdens and enhance their quality of life.

URL：https://www.sysmex.co.jp/en/index.html