Primary Biliary Cholangitis and Primary Sclerosing Cholangitis Therapy Landscape

Am J Gastroenterol. 2024 Oct 31. doi: 10.14309/ajg.0000000000003174. Online ahead of print.

Abstract

Primary biliary cholangitis (PBC) and primary sclerosing cholangitis (PSC) are rare, 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 (UDCA) has historically been the first-line treatment for PBC, with obeticholic acid (OCA) and fibrates used as second-line or adjunctive therapies. However, the treatment landscape is rapidly expanding. Recently, two new second line agents gained FDA approval for the treatment of PBC, and several other therapies remain under investigation with promising results. While significant progress has been made in development of therapies for PBC, there are no current approved treatments for PSC other than liver transplantation although several emerging therapies have shown encouraging results. This review outlines the current and upcoming treatments for PBC and PSC.