Obeticholic Acid for Primary Biliary Cholangitis

Primary biliary cholangitis (PBC) is a rare autoimmune cholestatic liver disease that may progress to fibrosis and/or cirrhosis. Treatment options are currently limited. The first-line therapy for this disease is the drug ursodeoxycholic acid (UDCA), which has been proven to normalize serum markers of liver dysfunction, halt histologic disease progression, and lead to a prolongation of transplant-free survival. However, 30-40% of patients unfortunately do not respond to this first-line therapy. Obeticholic acid (OCA) is the only registered agent for second-line treatment in UDCA-non responders. In this review, we focus on the pharmacological features of OCA, describing its mechanism of action of and its tolerability and efficacy in PBC patients. We also highlight current perspectives on future therapies for this condition.

Automated summary

Primary biliary cholangitis (PBC) is a rare autoimmune liver disease with limited treatment options. Ursodeoxycholic acid (UDCA) is the first-line therapy but is ineffective for a significant portion of patients. Obeticholic acid (OCA) is an effective second-line treatment for UDCA-non responders. Future therapies for PBC are promising.