The US Food and Drug Administration has approved durvalumab in combination with gemcitabine and cisplatin to treat adult patients with locally advanced or metastatic biliary tract cancer. Durvalumab, a drug approved for treating lung cancer, is an immune checkpoint inhibitor that targets programmed cell death-ligand 1 (PD-L1). The approval for the new indication of biliary tract cancer came after the global health agency reviewed the TOPAZ-1 trial. Earlier this year, the findings of the trial were presented at the Gastrointestinal Cancers Symposium organized by the American Society of Clinical Oncology (ASCO).

According to the study′s lead author, Dr. Do-Youn Oh, MD, Ph.D., professor in the Division of Medical Oncology at Seoul National University Hospital and Seoul National University College of Medicine in Korea, adding immunotherapy to standard chemotherapy can increase survival in biliary tract cancer without causing any new serious side effects.

The clinical trial included 685 patients, out of which 56% had intrahepatic cholangiocarcinoma, 25% had gallbladder cancer, and 19% had extrahepatic cholangiocarcinoma with varied demographics, such as 56% Asian people, 37% White people, 2% Black people, and 4% other races; 7% Hispanic or Latino people; 50% men and 50% women; median age, 64 years (range, 20-85 years); and 47% aged 65 years or older.

The results of the trial showed a statistically significant improvement in overall survival for patients who received immunotherapy in addition to chemotherapy, i.e., overall survival was 12.8 months with durvalumab vs 11.1 months with placebo. Similarly, the median progression-free survival and overall response rate were 7.2 months and 27% with durvalumab in comparison to 5.7 months and 19% with placebo, respectively. The most common adverse reactions observed during the trial period were fatigue, nausea, constipation, decreased appetite, abdominal pain, rash, and pyrexia. 

(Source: https://www.medscape.com/viewarticle/980227 )