Study Background
Primary biliary cholangitis (PBC) is a chronic autoimmune liver disease characterized by progressive destruction of intrahepatic bile ducts leading to cholestasis, fibrosis, and potentially cirrhosis and liver failure. Ursodeoxycholic acid (UDCA) is the standard first-line treatment; however, a significant subset of patients exhibits inadequate biochemical response, necessitating second-line therapies. Obeticholic acid (OCA), a farnesoid X receptor agonist, was conditionally approved to improve outcomes in UDCA non-responders or intolerant patients. Despite promising results from initial clinical trials, including the POISE study, concerns remain about OCA’s efficacy in reducing long-term liver-related clinical events and its safety profile, notably pruritus and use in cirrhotic populations. Regulatory actions like the European Medicines Agency’s (EMA) withdrawal of marketing authorization underscore the need for further real-world evidence.
