New AstraZeneca treatment for rare cancer reduces risk of death

AstraZeneca’s durvalumab – also known as Imfinzi – has shown statistically significant improvements in the overall survival of patients treated for biliary tract cancer (BTC), a rare and aggressive group of cancers.

During TOPAZ-1, the phase 3 clinical trial testing the safety and effectiveness of Imfinzi combined with chemotherapy, risk of death was reduced by 20% in patients with advanced BTC. Furthermore, it was the first phase 3 trial to show improved survival with an immunotherapy combination in this setting.

BTC is a spectrum of rare and aggressive gastrointestinal cancers that form in the cells of the bile ducts, gallbladder or ampulla of Vater. The ampulla of Vater is a small opening that enters into the first portion of the small intestine, where the bile duct and pancreatic duct connect to the small intestine.

Around 50,000 people in the US, Europe and Japan and about 210,000 people worldwide are diagnosed with BTC each year. Historically, it often has poor outcomes, with approximately 5% to 15% of all patients surviving five years.

“After minimal progress for more than a decade in advanced biliary tract cancer, the TOPAZ-1 results are a tremendous advance for our patients, showing a clear survival benefit for Imfinzi,” said Do-Youn Oh, principal investigator in the TOPAZ-1 phase 3 trial. “This combination will provide a desperately needed and potentially practice-changing new treatment option in a setting where the current prognosis is devastating.”

The trial has demonstrated an overall 25% reduction in the risk of disease progression and death when compared with chemotherapy alone. It has also delivered statistically significant and clinically meaningful results for patients with this rare and aggressive group of cancers, while also providing considerable hope for the future.