Positive results from the TOPAZ-1 clinical trial show that Imfinzi (durvalumab), in combination with standard-of-care chemotherapy, demonstrated a statistically significant and clinically meaningful overall survival benefit when compared to standard chemotherapy as a 1st-line treatment for patients with advanced biliary tract cancer.
Approximately 50,000 people in the US, Europe and Japan and about 210,000 people worldwide are diagnosed with biliary tract cancers each year.3-5 These patients have a poor prognosis, with approximately only 5% to 15% of all patients surviving five years.4 In December 2020, Imfinzi was granted Orphan Drug Designation in the US for the treatment of these cancers. Patients with advanced biliary tract cancer are in need of new treatments as progress in the 1st-line setting has remained largely stagnant for more than 10 years. The TOPAZ-1 clinical trial is the first to show that adding an immunotherapy to standard chemotherapy delivers a meaningful overall survival benefit.
About Biliary Tract Cancer
Biliary tract cancers are a group of rare and aggressive gastrointestinal cancers that form in the cells of the bile ducts (cholangiocarcinoma), gallbladder or ampulla of Vater (where the bile duct and pancreatic duct connect to the small intestine).1,2 Cholangiocarcinoma is more common in China and Thailand and is on the rise in Western countries.8-10 Gallbladder cancer is more common in certain regions of South America, India and Japan.11,12
Fyarro for Malignant Perivascular Epithelioid Cell Tumor (PEComa)
The U.S. Food and Drug Administration (FDA) has approved FYARRO™ (sirolimus protein-bound particles for injectable suspension) (albumin-bound) for intravenous use for the treatment of adult patients with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor (PEComa).
Radiofrequency Ablation Effective For Small Kidney Cancers
Radiofrequency ablation an an effective treatment for patients with small kidney cancers.
Imfinzi is a human monoclonal antibody directed against programmed death ligand-1 (PD-L1). PD-L1 can be expressed by tumors to evade detection by the immune system through binding to PD-1 on cytotoxic T lymphocytes. Imfinzi blocks the PD-L1 interaction with PD-1, countering the tumor’s immune-evading tactics. Imfinzi is being developed, alongside other immunotherapies, to empower the patient’s immune system and attack the cancer. Imfinzi is already FDA approved for certain patients with lung and bladder cancer.
About The TOPAZ-1 Clinical Trial
The TOPAZ-1 clinical trial directly compared standard first line gemcitabine plus cisplatin to the combination plus Imfinzi immunotherapy as a 1st-line treatment in 685 patients with unresectable advanced or metastatic disease including intrahepatic and extrahepatic cholangiocarcinoma, and gallbladder cancer (ampullary carcinoma was excluded). The trial is being conducted in more than 145 centers across 17 countries including in the US, Europe, South America and several countries in Asia including South Korea, Thailand, Japan, Taiwan and China. The data will be presented at a forthcoming medical meeting and shared with health authorities.
- Marcano-Bonilla L, et al. Biliary tract cancers: epidemiology, molecular pathogenesis and genetic risk associations. CCO. 2016;5(5).
- ESMO. What is Biliary Tract Cancer. Available here. Accessed October 2021.
- Siegel RL, Cancer statistics, 2020. CA Cancer J Clin 2020;70:7-30.
- ECIS - European Cancer Information System. Available here. Accessed October 2021.
- Kohei Nakachi, et al. Hepatobiliary and Pancreatic Oncology Group of the Japan Clinical Oncology Group, A randomized Phase III trial of adjuvant S-1 therapy vs. observation alone in resected biliary tract cancer: Japan Clinical Oncology Group Study (JCOG1202, ASCOT), Japanese Journal of Clinical Oncology. 2018,48:392-395.
- GBD 2017 Disease and Injury Incidence and Prevalence Collaborators. Global, regional, and national incidence, prevalence, and years lived with disability for 354 diseases and injuries for 195 countries and territories, 1990-2017: a systematic analysis for the Global Burden of Disease Study 2017. Lancet. 2018;392(10159):1789-1858.
- Turkes F, et al. Contemporary Tailored Oncology Treatment of Biliary Tract Cancers. Gastroenterol Res Pract. 2019;2019:7698786.
- Banales JM, Cardinale V, Carpino G, et al. Cholangiocarcinoma: current knowledge and future perspectives consensus statement from European network for the study of cholangiocarcinoma (ENS-CCA). Nat Rev Gastroenterol Hepatol. 2016;13:261-280.
- Kirstein MM, Vogel A. Epidemiology and risk factors of cholangiocarcinoma. Visc Med. 2016;32:395-400.
- Khan SA, Tavolari S, Brandi G. Cholangiocarcinoma: epidemiology and risk factors. Liver International. 2019;39(Suppl.1):19-31.
- Bridgewater JA, Goodman KA, Kalyan A, et al. Biliary tract cancer: epidemiology, radiotherapy, and molecular profiling. Am Soc Clin Oncol Educ Book. 2016;35:194-203.
- Torre LA, Siegel RL, Islami F, et al. Worldwide burden of and trends in mortality from gallbladder and other biliary tract cancers. Clin Gastroenterol Hepatol. 2018;16:427-437.
- Banales JM, et al. Cholangiocarcinoma 2020: the next horizon in mechanisms and management. Nature Reviews Gastroenterology & Hepatology. 2020; 17: 557-588.
- He XD, et al. Association of metabolic syndromes and risk factors with ampullary tumors development: A case-control study in China. World J Gastroenterol. 2014; 20(28): 9541-9548.
- WHO. World Cancer Fact Sheet. Available here. Accessed October 2021.