Merck KGaA Gets FDA Orphan Drug Tag for Biliary Tract Cancer Immunotherapy

• FDA grants M7824, an investigational bifunctional immunotherapy, orphan drug designation in biliary tract cancer
• First regulatory designation for M7824 following recent presentation of first clinical data in BTC
• BTC is a group of rare, aggressive gastrointestinal cancers associated with limited treatment options and poor outcomes

Merck KGaA, Darmstadt, Germany,  a leading science and technology company, which operates its healthcare business in the U.S. and Canada as EMD Serono, today announced that the US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to M7824, the first regulatory designation for the bifunctional immunotherapy, for the treatment of biliary tract cancer (BTC). The FDA orphan drug designation follows the recent presentation of the first clinical data for M7824 in BTC at the European Society of Medical Oncology (ESMO) congress in October. M7824 is an investigational bifunctional immunotherapy designed to combine co-localized blocking of the transforming growth factor-β and anti-PD-L1 immune escape mechanisms.

BTC is a collective term for a group of rare and aggressive gastrointestinal cancers, including intrahepatic cholangiocarcinoma (ICC), extrahepatic cholangiocarcinoma (ECC), and gallbladder carcinoma (GBC).^[1] Approximately 16,000 cases of BTC are estimated to occur every year in the US and collectively these cancers present late in the majority of patients. ^[1],[2] Treatment options are limited and the median survival rate in the advanced setting is less than one year, objective tumor response with commonly used chemotherapy is typically less than 10% with short duration of response. ^[1],[3],[5]  

“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,” said Luciano Rossetti, Head of Global Research & Development at the Biopharma business of Merck KGaA, Darmstadt, Germany. “As the first regulatory designation for M7824, Merck KGaA, Darmstadt, Germany is excited about the potential of this new class of immunotherapy in a number of challenging cancers and settings.”

The first clinical data for M7824 in BTC, presented at the ESMO congress in October, demonstrated clinical activity in Asian patients who had progressed after platinum-based first-line treatment. The ORR among the total of 30 patients was 20%, as assessed by IRC, and responses were observed across PD-L1 levels with a duration of response ranging from 8.3 months to 13.9+ months. Grade 3 or higher TRAEs were experienced by 10 patients (33.3%) and the most common Grade 3 TRAEs were rash (10%) and lipase increase (10%).

https://www.biospace.com/article/releases/merck-kgaa-darmstadt-germany-announces-fda-orphan-drug-designation-for-bifunctional-immunotherapy-m7824-in-biliary-tract-cancer/