Author information

• ¹Department of Gastroenterology and Hepatology, University Hospital Ku Leuven, Centre of ERN RARE-LIVER, Belgium. Electronic address: Frederik.nevens@uzleuven.be.
• ²Division of Gastroenterology and Hepatology, Department of Medicine III, Medical University of Vienna, Centre of ERN RARE-LIVER, Austria.
• ³Hannover Medical School, Centre of ERN RARE-LIVER, Hannover, Germany.

Abstract

The discovery of nuclear receptors and transporters has contribute to the development of new drugs for the treatment of cholestatic liver diseases. Especially progress has been made in the second line therapy of PBC. These new drugs can be separated into compounds primarily targeting cholestasis, molecules targeting fibrogenesis and molecules with immune-mediated action. Finally, also drugs aimed at symptom relief ( pruritus and fatigue) are further under investigation. Obeticholic acid is currently the only approved second line therapy for PBC. Drugs in the late phase of clinical development are the PPAR agonists, NorUDCA and the NOX 1&4 inhibitors.