FXR: structures, biology, and drug development for NASH and fibrosis diseases

Acta Pharmacol Sin. 2022 May;43(5):1120-1132. doi: 10.1038/s41401-021-00849-4. Epub 2022 Feb 25.

Abstract

The nuclear receptor farnesoid-X-receptor (FXR) plays an essential role in bile acid, glucose, and lipid homeostasis. In the last two decades, several diseases, such as obesity, type 2 diabetes, nonalcoholic fatty liver disease, cholestasis, and chronic inflammatory diseases of the liver and intestine, have been revealed to be associated with alterations in FXR functions. FXR has become a promising therapeutic drug target, particularly for enterohepatic diseases. Despite the large number of FXR modulators reported, only obeticholic acid (OCA) has been approved for primary biliary cholangitis (PBC) therapy as FXR modulator. In this review, we summarize the structure and function of FXR, the development of FXR modulators, and the structure-activity relationships of FXR modulators. Based on the structural analysis, we discuss potential strategies for developing future therapeutic FXR modulators to overcome current limitations, providing new perspectives for enterohepatic and metabolic diseases treatment.

Keywords: FXR modulators; farnesoid X receptor; liver fibrosis diseases; nuclear receptor; rational drug design; structure–activity relationships.

Publication types

  • Review

MeSH terms

  • Biology
  • Diabetes Mellitus, Type 2* / drug therapy
  • Drug Development
  • Fibrosis
  • Humans
  • Non-alcoholic Fatty Liver Disease* / drug therapy
  • Non-alcoholic Fatty Liver Disease* / metabolism