Multiple therapeutic targets in rare cholestatic liver diseases: Time to redefine treatment strategies

Abstract

Primary biliary cholangitis and primary sclerosing cholangitis are rare diseases affecting the bile ducts and the liver. The limited knowledge of their pathogenesis leads to limited therapeutic options. Nevertheless, the landscape of novel therapies for these cholangiopathies is now rapidly changing, providing new treatment opportunities for patients and clinicians involved in their care. The aim of this review is to summarize the evidence of novel molecules under investigation for primary biliary cholangitis and primary sclerosing cholangitis and to discuss how they can potentially change current treatment paradigms.

Keywords: Bile acids; FXR agonists; Fibrates; Gut–liver axis; Microbiome; Precision medicine; Primary biliary cholangitis; Primary sclerosing cholangitis.