Advances in pharmacotherapy for primary biliary cirrhosis

Hani S. Mousa, Ana Lleo, Pietro Invernizzi, Christopher L. Bowlus, Merril Eric Gershwin

Research output: Contribution to journalArticlepeer-review


Introduction: Primary biliary cirrhosis (PBC) is a chronic autoimmune liver disease mostly seen in middle-aged women characterized by progressive nonsuppurative destruction of small bile ducts resulting in intrahepatic cholestasis, parenchymal injury and ultimately end-stage liver disease. Despite major breakthroughs in our understanding of PBC, there remains only one FDA-approved agent for treatment: ursodeoxycholic acid (UDCA) to which one-third of patients are unresponsive. Areas covered: Biochemical response to treatment with UDCA is associated with excellent survival rates in PBC patients. However, there is a need for alternative treatments for nonresponders. Results from human epidemiological and genetic studies as well as preclinical studies in PBC animal models have provided a strong impetus for the development of new therapeutic agents. In this review, we discuss the recent advances in translational research in PBC focusing on promising therapeutic approaches, namely immune-based targeted therapies and agents targeting the synthesis and circulation of bile acids. Expert opinion: We are in a new era for the development of novel therapies for PBC. Data on fibrates, budesonide and obeticholic acid offer encouragement for nonresponders to UDCA.

Original languageEnglish
Pages (from-to)633-643
Number of pages11
JournalExpert Opinion on Pharmacotherapy
Issue number5
Publication statusPublished - Apr 1 2015


  • Biologics
  • Farnesoid X receptor agonists
  • Primary biliary cirrhosis
  • Ursodeoxycholic acid

ASJC Scopus subject areas

  • Pharmacology (medical)
  • Pharmacology
  • Medicine(all)


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