News Releases

Taiho Pharmaceutical Co., Ltd. (hereinafter “Taiho”) announced today that it has been granted approval from the Japanese Ministry of Health, Labour and Welfare to manufacture and market the FGFR inhibitor “LYTGOBI_® tablets 4mg” (generic name: futibatinib) for unresectable biliary tract cancer harboring FGFR2 gene fusions that has progressed after chemotherapy.

This approval is based on the data from the FOENIX-CCA2 trial. The 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 LYTGOBI 20mg once daily until disease progression or unacceptable toxicity. The trial’s primary endpoint was objective response rate (ORR)* based on independent review.
*ORR (objective response rate) : Percentage of patients with objective evidence of response to anticancer treatment and other treatments

Results from the trial were published in the January 19, 2023 issue of The New England Journal of Medicine.¹

Furthermore, a diagnostic device called the “OncoGuide™ NCC Oncopanel System” which detects FGFR2 gene fusions, co-developed with Sysmex Corporation, obtained additional approval for a partial change as a companion diagnostic to LYTGOBI in February 2023 in Japan.

Taiho believes that LYTGOBI will make a positive contribution as a new treatment option for patients with biliary tract cancer.

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.²

LYTGOBI (development code: TAS-120), discovered by Taiho, 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 biliary tract cancer, who were previously treated with chemotherapy or other therapies. LYTGOBI 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

The U.S. Food and Drug Administration (FDA) approved LYTGOBI for the treatment of adult patients with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma (iCCA) harboring FGFR2 gene fusions or other rearrangements in September 2022.

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 LYTGOBI for the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma (CCA) with a FGFR2 fusion or rearrangement that have progressed after at least one prior line of systemic therapy.

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

1 L. Goyal, F. Meric-Bernstam, A. Hollebecque, et al. Futibatinib for FGFR2-Rearranged Intrahepatic Cholangiocarcinoma, N Engl J Med 2023;388:228-39.
2 Calculated based on National Cancer Institute Cancer Information Service, Cancer Statistics (National Cancer Registry). National Cancer Incidence Data (2016-2018) and Follow-up Survey Committee, Liver Cancer Study Group of Japan. Kudo M, Izumi N, Kubo S, Kunido N, Sakamoto T, et al. The 21st National Primary Liver Cancer Follow-up Study Report (2010-2011). Liver. 2020;61:645-691.