Obeticholic acid for the treatment of primary biliary cirrhosis

Kidist K. Yimam, Christopher Bowlus

Research output: Contribution to journalArticlepeer-review

4 Scopus citations


Introduction: Primary biliary cirrhosis (PBC) is a rare autoimmune liver disease, in which the epithelial cells of small bile ducts are targeted for immune attack leading to cholestasis and cirrhosis. Although it is considered an autoimmune disease, immune modulators have not been effective to date in the treatment of PBC. Ursodeoxycholic acid (UDCA) has been the mainstay of treatment of over two decades, but a significant minority of PBC patients do not have a complete response to UDCA and are at risk of continued disease progression.Area covered: Obeticholic acid (OCA) is a novel derivative of chenodeoxycholic acid, a primary human bile acid and natural ligand of the nuclear hormone receptor farnesoid X receptor (FXR). Activation of FXR inhibits bile acid (BA) synthesis and protects against the toxic accumulation of BAs. We reviewed published preclinical, efficacy, and safety data of OCA for the treatment of PBC.Expert opinion: OCA in combination with UDCA leads to significant improvement in liver biochemistries shown to predict outcomes in PBC patients with an incomplete response to UDCA. Dose-dependent pruritus is the most common adverse effect. Long-term efficacy of OCA and its efficacy in patients with advanced PBC need to be delineated, as well as its role as monotherapy compared with UDCA.

Original languageEnglish (US)
Pages (from-to)1351-1358
Number of pages8
JournalExpert Opinion on Orphan Drugs
Issue number12
StatePublished - Dec 1 2014


  • Bile acid
  • Chenodeoxycholic acid
  • Farnesoid X receptor
  • Nuclear receptors
  • Obeticholic acid
  • Primary biliary cirrhosis

ASJC Scopus subject areas

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


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