December 21, 2022: “AstraZeneca’s Imfinzi (durvalumab) has been approved in the European Union (EU) for the 1st-line treatment of adult patients with unresectable or metastatic biliary tract cancer (BTC) in combination with chemotherapy (gemcitabine plus cisplatin).
The approval by the European Commission was based 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.
The approval follows the recommendation by The Committee for Medicinal Products for Human Use of the European Medicines Agency in November 2022.
At the interim analysis, 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).
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 treated with Imfinzi plus chemotherapy 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 (cholangiocarcinoma) and gallbladder.
There are approximately 211,000 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 and The Christie NHS Foundation Trust, UK, and a lead investigator in the TOPAZ-1 Phase III trial, said: “Today’s approval marks an important shift in the treatment of this aggressive and often overlooked disease and a significant improvement compared to standard of care for these patients.
After waiting over a decade for new therapeutic options, biliary tract cancer patients in the EU will now have the opportunity to benefit from an immunotherapy-based treatment for the first time.”
Dave Fredrickson, Executive Vice President, Oncology Business Unit, AstraZeneca, said: “With this approval, Imfinzi plus chemotherapy becomes the only immunotherapy-based treatment option available to patients in the EU with advanced biliary tract cancer.
This approval underscores our commitment to transform survival outcomes while addressing the high unmet need for new and improved treatments for patients with hepatobiliary cancers.”
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 plus chemotherapy, and by 63.5% of patients treated with chemotherapy alone.
Imfinzi plus chemotherapy is approved in the US and other countries for the treatment of adults 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.”