Imfinzi + chemotherapy approved in China as first immunotherapy regimen for patients with locally advanced or metastatic biliary tract cancer

The approval by China’s National Medical Products Administration (NMPA) was based on the primary results from the TOPAZ-1 Phase III trial published in the New England Journal of Medicine Evidence (previously cited), as well as a prespecified exploratory analysis of an additional cohort of patients in China.

BTC is a group of rare and aggressive cancers that occur in the bile ducts (cholangiocarcinoma) and gallbladder. An estimated 211,000 new patients are diagnosed with gallbladder and biliary tract cancer each year, and nearly one in five patients diagnosed is in China. These patients have a poor prognosis, with approximately 5% to 15% of patients with BTC surviving five years.

In an interim analysis of the TOPAZ-1 Phase III trial, Imfinzi plus chemotherapy reduced the risk of death by 20% versus chemotherapy alone (based on a hazard ratio [HR] of 0.80; 95% confidence interval [CI] 0.66-0.97; p=0.021). Median OS was 12.8 months versus 11.5 for chemotherapy. Additionally, efficacy results from a prespecified exploratory analysis in an additional cohort of TOPAZ-1 patients enrolled in China were consistent with those in the overall global trial population, showing Imfinzi plus chemotherapy reduced the risk of death by 22% versus chemotherapy alone (HR of 0.78; 95% CI, 0.51-1.18).