Targets and investigative treatments for primary biliary cholangitis

Ahmad H. Ali, Elizabeth J. Carey, Keith D. Lindor

Research output: Contribution to journalReview articlepeer-review


Introduction: Primary biliary cholangitis (PBC) is an autoimmune disease of the liver that can slowly progress to cirrhosis, end-stage liver disease and its consequent complications. Areas covered: Ursodeoxycholic acid (UDCA) is approved for the treatment of PBC at a daily dose of 13 to 15 mg/kg/day, and should be continued indefinitely unless intolerance to UDCA occurs. Nearly 40% of patients have inadequate response to UDCA; therefore, there is a compelling need for newer and more effective therapies. Research has led to the discovery of several novel pathways that are thought to be implicated in the pathogenesis of PBC. Phase II and III clinical trials of obeticholic acid (OCA), the leading farnesoid X receptor agonist compound, in PBC patients with inadequate response to UDCA have shown that the use of OCA results in significant improvement in liver biochemistries. OCA has been recently recommended by the FDA’s Advisory Board for approval for treatment of PBC. Expert opinion: PBC remains a medical and socioeconomic problem. UDCA is currently the only medication approved for treatment of PBC. The discovery of novel therapeutic targets in PBC will hopefully lead to better outcomes in PBC.

Original languageEnglish (US)
Pages (from-to)1011-1020
Number of pages10
JournalExpert Opinion on Orphan Drugs
Issue number10
StatePublished - Oct 2 2016


  • Primary biliary cholangitis
  • antimitochondrial antibody
  • cirrhosis
  • end-stage liver disease
  • farnesoid X receptor
  • liver transplantation
  • obeticholic acid
  • ursodeoxycholic acid

ASJC Scopus subject areas

  • Pharmacology, Toxicology and Pharmaceutics (miscellaneous)
  • Health Policy
  • Pharmacology (medical)


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