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FDA grants orphan drug designation to M7824 for biliary tract cancer
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The FDA granted orphan drug designation to M7824 for the treatment of biliary tract cancer.
This is the first regulatory designation for M7824 (Merck/EMD Serono), an investigational bifunctional immunotherapy designed to combine co-localized blocking of the transforming growth factor-beta and anti-PD-L1 immune escape mechanisms.
Biliary tract cancer is the collective term for a group of rare, aggressive malignancies, including intrahepatic cholangiocarcinoma, extrahepatic cholangiocarcinoma and gallbladder carcinoma.
Limited treatment options exist and outcomes often are poor. Patients often present with late-stage disease and median survival for those with advanced disease typically is less than a year.
A study presented in October at European Society for Medical Oncology Congress showed M7824 demonstrated clinical activity among Asian patients who progressed after platinum-based first-line treatment.
The study included 30 patients. Researchers reported a 20% objective response rate as assessed by independent review committee, with responses observed across PD-L1 levels. Durations of response ranged from 8.3 months to more than 13.9 months.
Ten patients (33.3%) experienced grade 3 or higher treatment-related adverse events, the most common of which were rash and lipase increase (10% each).
“Biliary tract cancer is a rare, notoriously hard-to-treat tumor where existing treatment approaches — such as surgery or chemotherapy — are either not viable or simply don’t deliver acceptable patient outcomes,” Luciano Rossetti, head of global research and development at the biopharma business of Merck KGaA, Darmstadt, Germany, said in a company-issued press release. “[The company] is excited about the potential of this new class of immunotherapy in a number of challenging cancers and settings.”
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.