Primary Biliary Cholangitis and Primary Sclerosing Cholangitis Therapy Landscape

Am J Gastroenterol. 2025 Jan 1;120(1):151-158. doi: 10.14309/ajg.0000000000003174. Epub 2024 Oct 31.

Abstract

Primary biliary cholangitis (PBC) and primary sclerosing cholangitis (PSC) are rare, and chronic cholestatic diseases that can progress to liver failure. The goals of treatment are to halt the progression of liver disease to cirrhosis and/or liver failure, and alleviate symptoms associated with these diseases. Ursodeoxycholic acid has historically been the first-line treatment of PBC, with obeticholic acid and fibrates used as second-line or adjunctive therapies. However, the treatment landscape is rapidly expanding. Recently, 2 new second-line agents gained US Food and Drug Administration approval for the treatment of PBC, and several other therapies remain under investigation with promising results. Although significant progress has been made in the development of therapies for PBC, there are no current approved treatments of PSC other than liver transplantation although several emerging therapies have shown encouraging results. This review outlines the current and upcoming treatments of PBC and PSC.

Publication types

  • Review

MeSH terms

  • Chenodeoxycholic Acid* / analogs & derivatives
  • Chenodeoxycholic Acid* / therapeutic use
  • Cholagogues and Choleretics* / therapeutic use
  • Cholangitis, Sclerosing* / complications
  • Cholangitis, Sclerosing* / drug therapy
  • Cholangitis, Sclerosing* / therapy
  • Fibric Acids / therapeutic use
  • Humans
  • Liver Cirrhosis, Biliary* / complications
  • Liver Cirrhosis, Biliary* / drug therapy
  • Liver Cirrhosis, Biliary* / therapy
  • Liver Transplantation
  • Ursodeoxycholic Acid* / therapeutic use

Substances

  • Ursodeoxycholic Acid
  • Chenodeoxycholic Acid
  • obeticholic acid
  • Cholagogues and Choleretics
  • Fibric Acids