M7824 Receives Orphan Drug Designation for Biliary Tract Cancer

BTC Is a Group of Rare Gastrointestinal Cancers With Limited Treatment Options, Poor Outcomes

The FDA has granted orphan drug status to M7824 (Merck KGaA, Darmstadt, Germany) for the treatment of biliary tract cancer (BTC). M7824 is an investigational bifunctional immunotherapy that combines a TGF-β trap with the anti-PD-L1 mechanism in one fusion protein.

BTC, a group of rare and aggressive gastrointestinal cancers, includes intrahepatic cholangiocarcinoma (ICC), extrahepatic cholangiocarcinoma (ECC), and gallbladder carcinoma (GBC). Each year in the U.S., around 16,000 cases of BTC are estimated to occur, and these cancers typically present late in most patients. BTC is notoriously hard to treat and surgery or chemotherapy are seldom viable. The median survival rate in the advanced setting is less than one year.

Clinical data for M7824 demonstrated clinical activity in patients who had progressed after platinum-based first-line treatment. The overall response rate among the total of 30 patients was 20% and responses were observed across PD-L1 levels with a duration of response ranging from 8.3 months to 13.9+ months.

M7824 is a vital part of a novel combination approach aiming to harness the immune system’s power and address the extremely intricate nature of difficult-to-treat tumors. It is currently in phase I studies for solid tumors, and a trial to investigate M7824 compared with pembrolizumab as a first-line treatment in patients with PD-L1 expressing advanced NSCLC.

Source: PR Newswire, December 10, 2018