Durvalumab With Chemo Wins FDA Approval in Treatment of Biliary Tract Cancers

An estimated 1 in 4 patients treated with durvalumab and chemotherapy (gemcitabine plus cisplatin) was alive at 2 years compared with 1 in 10 treated with chemotherapy alone, with these results contributing to the FDA's approval of the first immunotherapy to treat these cancers.

The FDA has approved durvalumab in combination with chemotherapy to treat adult patients with locally advanced or metastatic biliary tract cancer, making durvalumab the first immunotherapy approved for these rare cancers with poor survival rates.

The approval, announced in a statement Monday by AstraZeneca, which markets durvalumab as Imfinzi, was based on the results of the phase 3 TOPAZ-1 trial. An interim analysis of TOPAZ-1 plus chemotherapy found that the combination reduced the risk of death by 20% compared with chemotherapy alone (HR, 0.80; 95% CI, 0.66-0.97; P = .021).

An estimated 1 in 4 patients treated with durvalumab and chemotherapy (gemcitabine plus cisplatin) was alive at 2 years compared with 1 in 10 treated with chemotherapy alone. According to Monday’s statement, investigators found results were consistent across subgroups, regardless of programmed death ligand-1 expression or tumor location, which can be a key factor in treatment. Surgery to remove the tumor is indicated in many cases, but is not always possible.

About 23,000 people in the United States receive a biliary tract cancer diagnosis each year. While still rare, these cancers are among the cancers that are increasing among those age 45 or younger in Western countries, possibly due to rising rates of obesity. Historically, overall survival rates across all age groups are poor, with 5-year rates between 5% and 15%.

Results for TOPAZ-1 were first revealed in January during the American Society of Clinical Oncology Gastrointestinal Cancers Symposium and published in the New England Journal of Medicine. Follow-up results are to be presented at the upcoming European Society for Medical Congress in Paris, France, which takes place September 9-13.

While there have been advances in this group of cancers in recent years, they have involved targeted treatments for patients with specific mutations, with approvals seen in second-line treatment. This advance will change the standard of care in first-line treatment, and the durvalumab combination has been added to the National Comprehensive Cancer Network guidelines.

“This approval represents a major step forward for patients with advanced biliary tract cancer, who urgently need new, well-tolerated, and effective treatment options after more than a decade of limited innovation,” Aiwu Ruth He, MD, PhD, associate professor of medicine, leader of the GI Cancer Program, Georgetown Lombardi Comprehensive Cancer Center, Medstar Georgetown University Hospital, Washington, DC, and a lead investigator in the TOPAZ-1 trial, said in the statement.

“The combination of durvalumab and chemotherapy should become a new standard of care in this setting, having demonstrated significantly improved survival for these patients who have historically faced a poor prognosis.”

“Patients have been waiting a long time for a new, first-line treatment option for biliary tract cancer,” Stacie Lindsey, CEO of the Cholangiocarcinoma Foundation, said in the statement.