A Review of Therapies for Primary Biliary Cholangitis.

Abstract

Primary biliary cholangitis (PBC) is a rare, chronic, progressive autoimmune cholestatic liver disease that can lead to cirrhosis if left untreated. Ursodeoxycholic acid remains the standard first-line therapy; however, approximately 40% of patients exhibit an inadequate or partial biochemical response, and approximately 5% to 10% of patients are intolerant to the drug. Consequently, there remains a substantial need for effective second-line agents. Until recently, obeticholic acid (OCA) and fibrates were the only available second-line therapies. However, the therapeutic landscape for PBC has evolved significantly with the US Food and Drug Administration conditional approval of elafibranor and seladelpar as second-line therapies for patients without cirrhosis. Notably, OCA has been withdrawn as an available treatment option, further shifting the current treatment paradigm. This article aims to examine the current pharmacologic landscape of first-line and second-line PBC treatments, with an emphasis on clinical considerations and strategies for individualized treatment selection to optimize patient outcomes.