Maralixibat is in clinical development for the treatment of Progressive Familial Intrahepatic Cholestasis Type 2 (PFIC2). PFIC2 is an inherited condition that causes progressive liver disease, which normally leads to liver failure. This disease predominantly affects children. In PFIC2, bile acids, part of the fluid produced by the liver, which helps digestion, build up in liver cells, becoming toxic to the liver. If left untreated, this leads to end stage liver disease, increased risk of developing hepatocellular carcinoma (a form of liver cancer) and death. There are currently few effective treatments for PFIC patients and the condition can lead to the need for a liver transplant.
Maralixibat is an orally administered drug that is expected to reduce the level of bile acids. It is expected to interfere with the process by which most bile acids in the intestines are recovered and delivered back to the liver through the blood, thereby reducing the liver damage and itching seen in patients with PFIC2. If licensed, maralixibat will provide the first disease-modifying treatment option for patients with PFIC2, a disease of unmet clinical need.