News Releases

Taiho Pharmaceutical Co., Ltd. (“Taiho Pharmaceutical”) announced that its U.S. subsidiary, Taiho Oncology, Inc. (“Taiho Oncology”), has been granted U.S. Food and Drug Administration (FDA) approval of futibatinib (Development code: TAS-120, U.S, Product Name: LYTGOBI^®, Form: Tablets) 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. The indication is approved under accelerated approval^※1 based on overall response rate and duration of response in the FOENIX-CCA2 trial. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).

※1 Accelerated approval: The FDA instituted its Accelerated Approval program to allow for earlier approval of drugs that treat serious conditions, and that fill an unmet medical need based on a surrogate endpoint.

“Futibatinib is a new therapy option for patients with iCCA that can be taken orally,” said Tim Whitten, President and CEO of Taiho Oncology. “This approval is an important milestone for patients and may provide hope for improved outcomes. As someone whose family has been impacted by CCA, I’m acutely aware of the impact this disease can have on the patient and their loved ones.”

“Futibatinib is a key example of the potential of precision medicine in iCCA and represents another advance in the treatment of this rare and challenging disease,” said medical oncologist Lipika Goyal, MD, MPhil, of the Massachusetts General Hospital Cancer Center and lead investigator of the FOENIX-CCA2 trial. “As a researcher and a physician who treats patients with CCA, I am encouraged that treatment options continue to expand and evolve.”

Futibatinib was discovered by Taiho Pharmaceutical, which continues to develop this product together with Taiho Oncology, for other potential tumor types. “The Taiho group is working as one to optimize this agent for the patients who are waiting,” said Teruhiro Utsugi, Senior Managing Director at Taiho Pharmaceutical.

Through research and development of innovative treatments, the Taiho group aims to contribute to patients and healthcare professionals around the world.

(Intrahepatic, extrahepatic) Cholangiocarcinoma is diagnosed in approximately 8,000 individuals each year in the U.S.^ⅰ Approximately 20% of patients diagnosed with CCA have the intrahepatic form of the disease.^ⅱ、ⅲ Within this 20%, approximately 10-16% of patients have FGFR2 gene rearrangements, including fusions, which promote tumor proliferation.^{ⅳ、ⅴ、ⅵ、ⅶ、ⅷ}

Futibatinib (TAS-120) is an investigational, oral, potent, selective tyrosine kinase inhibitor of FGFR1, 2, 3 and 4. Futibatinib selectively 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. Futibatinib is being studied alone as a potential treatment for patients with advanced solid tumors with FGFR1-4 genetic aberrations, including CCA, or in combination with chemotherapy or other therapies.

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.

FOENIX-CCA2^※2 trial was a Phase 2 trial in 103 patients with locally advanced or metastatic unresectable iCCA 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) ^※3.

For details of the FOENIX-CCA2 trial, please refer to ClinicalTrials.gov ( https://clinicaltrials.gov/ct2/home trial ID：NCT02052778).

※2 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

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

Taiho Pharmaceutical, a subsidiary of Otsuka Holdings Co., Ltd. (https://www.otsuka.com/en/ ), is an R&D-driven specialty pharma focusing on the fields of oncology, allergy and immunology, and urology. Its corporate philosophy takes the form of a pledge: “We strive to improve human health and contribute to a society enriched by smiles.” In the field of oncology, in particular, Taiho Pharmaceutical is known as a leading company in Japan for developing innovative medicines for the treatment of cancer, a reputation that is rapidly expanding through their extensive global R&D efforts. In areas other than oncology, as well, the company creates and markets quality products that effectively treat medical conditions and can help improve people’s quality of life. Always putting customers first, Taiho Pharmaceutical also aims to offer consumer healthcare products that support people’s efforts to lead fulfilling and rewarding lives.
For more information about Taiho Pharmaceutical, please visit https://www.taiho.co.jp/en/

The mission of Taiho Oncology, Inc. is to improve the lives of patients with cancer, their families and their caregivers. The company specializes in the development of orally administered anti-cancer agents and markets these medicines for a range of tumor types in the U.S. Taiho Oncology’s growing pipeline of antimetabolic and selectively targeted anti-cancer agents is led by a world-class clinical development organization. Taiho Oncology is a subsidiary of Taiho Pharmaceutical Co., Ltd. which is part of Otsuka Holdings Co., Ltd. Taiho Oncology is headquartered in Princeton, New Jersey and oversees its parent company’s European and Canadian operations, which are located in Zug, Switzerland and Oakville, Ontario, Canada.

For more information, visit http://www.taihooncology.com

ⅰ: American Cancer Society. Key statistics for bile duct cancer. https://www.cancer.org/cancer/bile-duct-cancer/about/key-statistics.html#:~:text=Bile%20duct%20cancer%20(cholangiocarcinoma)%20is,diagnosed%20with%20it%20each%20year. Accessed July 2022.

ⅱ : Valle JW et al. Lancet. 2021;397:428-444.

ⅲ：Banales JM et al. Nat Rev Gastroenterol Hepatol. 2020;17:557-588.

ⅳ：Arai Y, Totoki Y, Hosoda F, et al. Fibroblast growth factor receptor 2 tyrosine kinase fusions define a unique molecular subtype of cholangiocarcinoma. Hepatology. Apr 2014;59(4):1427-34.10.1002/hep.26890.

ⅴ：Javle M, Bekaii-Saab T, Jain A, et al. Biliary cancer: Utility of next-generation sequencing for clinical management. Cancer. Dec 15 2016;122(24):3838-3847.10.1002/cncr.30254.

ⅵ：Sia D, Losic B, Moeini A, et al. Massive parallel sequencing uncovers actionable FGFR2-PPHLN1 fusion and ARAF mutations in intrahepatic cholangiocarcinoma. Nat Commun. Jan 22 2015;6:6087.10.1038/ncomms7087.

ⅶ：Silverman IM, Murugesan K, Lihou CF, et al. Comprehensive genomic profiling in FIGHT-202 reveals the landscape of actionable alterations in advanced cholangiocarcinoma. Journal of Clinical Oncology. 2019;37(15_suppl):4080-4080.10.1200/JCO.2019.37.15_suppl.4080.

ⅷ：Javle MM, Murugesan K, Shroff RT, et al. Profiling of 3,634 cholangiocarcinomas (CCA) to identify genomic alterations (GA), tumor mutational burden (TMB), and genomic loss of heterozygosity (gLOH). Journal of Clinical Oncology. 2019;37(15_suppl):4087-4087.10.1200/JCO.2019.37.15_suppl.4087.