Experimental Pharmacological Agents for the Treatment of Primary Biliary Cholangitis

Abstract

The standard therapy for primary biliary cholangitis (PBC) is ursodeoxycholic acid (UDCA) which has shown to improve hepatic biochemistry, delay histological progression and improve transplant-free survival. Approximately 30–40% of patients do not respond or are intolerant to UDCA. Obeticholic acid, a farnesoid X receptor (FXR) agonist is the only agent approved by the Food and Drug Administration for patients who do not respond to UDCA. Recently, combination therapy with UDCA and bezafibrate has been shown to improve biochemistry and both GLOBE and UK-PBC score in patients with an inadequate response to UDCA. More recently, new pharmacological agents have been included in Phase 2 and Phase 3 trials: PPAR agonists, non-bile acid FXR agonists, anti-NOX agents, immunomodulators and mesenchymal stem cells transplantation. This review gives an overview on the current experimental pharmacological agents employed in the treatment of PBC.

Keywords:

• PBC
• treatment
• ursodeoxycholic acid
• obeticholic acid
• fibrates
• FXR agonists
• PPAR agonists

Disclosure

Annarosa Floreani has received consultancy fees during the last two years from Intercept. The author reports no other conflicts of interest in this work.