Adding durvalumab (Imfinzi®) to chemotherapy significantly improves overall survival in patients with advanced biliary tract cancer, according to interim results from a phase 3 clinical trial called TOPAZ-1. The dual treatment also improved progression-free survival and overall response rate.

The combination was well-tolerated, and the discontinuation rate due to adverse events was not higher than that with chemotherapy alone.

Do-Youn Oh, MD, PhD, the principal investigator of the trial, stated “Patients with advanced biliary tract cancer are in dire need of new treatments as progress in the 1st-line setting has remained largely stagnant for more than 10 years.” Dr. Oh added, “TOPAZ-1 is the first Phase III trial to show that adding an immunotherapy to standard chemotherapy delivers a meaningful overall survival benefit for patients in this setting. Today’s exciting results are a major step forward in treating this disease and represent new hope for our patients.”

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TOPAZ-1 is a randomized, double-blind, placebo-controlled, multiregional, international study that aims to assess the safety and efficacy of durvalumab plus the chemotherapy agents gemcitabine and cisplatin compared to placebo plus the chemotherapy agents. 

Its primary endpoint is overall survival. The secondary endpoints are progression-free survival and overall response rate. 

The trial started on April 16, 2019, and it is still recruiting participants aged 18 years and older across the world with histologically confirmed advanced or metastatic biliary tract cancer that cannot be resected. Biliary tract cancer is a rare group of cancers with high mortality consisting of intrahepatic and extrahepatic cholangiocarcinoma and gallbladder carcinoma. It aims to recruit 757 participants and is estimated to be completed on June 30, 2022.

Read more about cholangiocarcinoma therapies

Durvalumab is a monoclonal antibody in humans that binds to programmed death-ligand 1 (PD-L1) protein and blocks its interaction with its receptor PD-1 and CD80 proteins, thereby releasing the inhibition of the immune response. It is already approved for the treatment of certain types of lung cancer and bladder cancer. In 2017, durvalumab received Orphan Drug Designation by the US Food and Drug Administration (FDA) for the treatment of biliary tract cancer.


Imfinzi plus chemotherapy significantly improved overall survival in 1st-line advanced biliary tract cancer in TOPAZ-1 phase III trial at interim analysis. News release. AstraZeneca; October 25, 2021.