Maralixibat chloride for Progressive familial intrahepatic cholestasis

Maralixibat is in clinical development for treating Progressive Familial Intrahepatic Cholestasis (PFIC). PFIC is the name given to a group of rare genetic disorders affecting the liver and impacting newborns, infants, and children. In PFIC, bile acids, which are produced by the liver to help digestion, build up in liver cells (cholestasis), causing liver disease. As a result of the cholestasis, patients can experience distressing symptoms such as pruritis (itching), jaundice, failure to thrive and a fat-soluble vitamin deficiency secondary to impaired absorption of fats. PFIC progresses at varying rates depending on the type. Patients may require liver transplantation due to intractable pruritus, impaired growth, or progressive liver disease with fibrosis usually developing into cirrhosis within the first decade of life. In severe cases, it can lead to end stage liver disease, increased risk of developing hepatocellular carcinoma (a form of liver cancer) and death.