Obeticholic acid (OCA) is a synthetic derivative of the endogenous primary bile acid, chenodeoxycholic acid, and is primarily used to treat liver and bile duct pathologies. It has been approved by the FDA for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA. OCA has also been studied for its potential use in treating non-alcoholic steatohepatitis (NASH).

The mechanism of action of OCA involves reducing hepatic exposure to bile acids, which limits the progression of cholangitic processes such as PBC and primary sclerosing cholangitis (PSC). OCA is a first-in-class agonist that selectively binds to the farnesoid X receptor (FXR). This leads to direct and indirect suppression of bile acid production in the liver and increased bile flow, resulting in reduced exposure of the liver to toxic levels of bile acids. This reduced exposure should clinically translate into decreased parenchymal liver injury caused by bile acids, leading to improved biochemical enzyme profiles and decreased disease progression of PBC.