Primary biliary cholangitis: From diagnosis to treatment

Primary biliary cholangitis (PBC), previously known as primary biliary cirrhosis and which has been designated an orphan condition, is a chronic autoimmune disease resulting in the destruction of the small bile ducts in the liver. Like other complex disorders, PBC is heterogeneous in its pre-sensation, symptomatology, disease progression and response to therapy. Without effective treatment, disease progression frequently leads to liver failure and death. Until May 2016, when Obeticholic acid (OCA) has been licensed as treatment options for patients with PBC, the only FDA-approved treatment for PBC was Ursodeoxycholic acid (UDCA), an oral hydrophilic bile acid, which can slow progression of liver damage due to PBC. However, 1 out of 3 patients taking UDCA has an inadequate biochemical response, leading to increased risk of disease progression, liver transplantation, and mortality. Given this unmet clinical need, new therapies are in development for the treatment of PBC. There are molecules targeting the immune-mediated response and bile-acid therapies targeting the bile acid biosynthetic and feedback processes that drive the biliary epithelial cells injury. In addition, antifibrotic therapies are under study to tackle the downstream damage following biliary injury. In conclusion, PBC is a condition with a heterogeneous, poorly characterized phenotype for which limited therapeutic options are currently available.