New therapies for primary biliary cholangitis usher in a personalised approach to treatment
摘要
Primary biliary cholangitis (PBC) is an autoimmune disease that damages the small to medium bile ducts of the liver. Left untreated, PBC leads to cirrhosis and liver failure. Until recently the only approved treatments for PBC were ursodeoxycholic acid (UDCA) and obeticholic acid, with fibrates used off-label. Common symptoms (pruritus and fatigue) are poorly managed by these treatments, and about 20–30% of people with PBC have an inadequate response to first-line treatment with UDCA. Ongoing drug development efforts have led to US Food and Drug Administration approval of elafibranor and seladelpar, two peroxisome proliferator-activated receptor agonists. The approval of new therapeutic agents is expected to enable a more personalised approach to treatment.