Obeticholic Acid for Treatment-Refractory Primary Biliary Cirrhosis

Ursodeoxycholic acid (UDCA) is currently the only therapy approved for treatment of primary biliary cirrhosis (PBC), but patients often have inadequate biochemical response to UDCA. In a double-blind, industry-funded trial, investigators tested the efficacy of obeticholic acid (OCA) — a potent farnesoid X receptor agonist with antifibrotic, anticholestatic, and anti-inflammatory properties — for PBC.

Participants were 165 patients (95% women) with alkaline phosphatase (ALP) level >1.5 times the upper limit of normal (i.e., inadequate biochemical response) despite 6 months of UDCA therapy. They were randomized to receive 10 mg, 25 mg, or 50 mg of OCA or placebo daily for 3 months. The primary endpoint was reduction in serum ALP level from bas…