AstraZeneca Imfinzi plus chemotherapy recommended for marketing authorisation in EU for advanced biliary tract cancer

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency based its positive opinion on the primary results from the TOPAZ-1 Phase III trial published in the New England Journal of Medicine Evidence, and on the updated results presented at the European Society for Medical Oncology Congress 2022.

At the interim analysis, Imfinzi plus chemotherapy reduced the risk of death by 20% versus chemotherapy alone (hazard ratio [HR] 0.80; 95% confidence interval [CI] 0.66-0.97; p=0.021).

Updated results from TOPAZ-1 after an additional 6.5 months of follow-up showed a 24% reduction in the risk of death versus chemotherapy alone (HR 0.76; 95% CI, 0.64-0.91), with more than two times as many patients estimated to be alive at two years versus chemotherapy alone (23.6% versus 11.5%). Updated median overall survival (OS) was 12.9 months versus 11.3 with chemotherapy.

BTC is a group of rare and aggressive cancers that occur in the bile ducts and gallbladder. There are approximately 210,000 new patients diagnosed with gallbladder and biliary tract cancer each year, and about 40,000 of these occur across Europe. These patients have a poor prognosis, with approximately 5% to 15% of patients with BTC surviving five years.

Juan W. Valle, MD, Professor of Medical Oncology at the University of Manchester, UK, and a lead investigator in the TOPAZ-1 Phase III trial, said: "This positive opinion is welcome news for patients with advanced biliary tract cancer in the European Union who face a poor prognosis and limited treatment options. The combination of durvalumab and chemotherapy is a significant advance for patients after more than a decade of limited progress, and this regimen should become a new standard of care option once approved."

Susan Galbraith, Executive Vice President, Oncology R&D, AstraZeneca, said: "If approved, Imfinzi plus chemotherapy will provide patients with advanced biliary tract cancer the first opportunity for treatment with an immunotherapy-based combination. This innovative regimen has been shown to significantly prolong patients' lives, and we look forward to bringing this option to those in the European Union as soon as possible."

Imfinzi plus chemotherapy was generally well tolerated, with no new safety signals observed, and did not increase the discontinuation rate due to adverse events (AEs) compared to chemotherapy alone. Grade 3 or 4 treatment-related AEs were experienced by 60.9% of patients treated with Imfinzi and chemotherapy, and by 63.5% of patients treated with chemotherapy alone.

Imfinzi plus chemotherapy is approved in the US, Canada, South Korea and Brazil for the treatment of patients with locally advanced or metastatic BTC. Regulatory applications are also currently under review in Japan and several other countries based on the TOPAZ-1 results.