Obeticholic acid (OCA) is a novel bile acid analogue that has shown positive effects on biochemical markers of liver function in phase II trials and was approved by the FDA for treatment of primary biliary cholangitis (PBC) after failure of ursodeoxycholic Acid (UDCA). Primary biliary cholangitis (PBC), which has until recently been called primary biliary cirrhosis, is a rare, chronic, progressive autoimmune liver disease that affects mainly middle-aged women. In the US, up to 130,000 individuals may have PBC.

In January 2015, obeticholic acid also received a breakthrough designation for treatment of non-alcoholic steatohepatitis (NASH) with concomitant liver fibrosis. Interim findings from the phase III trial are not expected to be available until March 2017; however, clinical interest in potential off-label use of obeticholic acid is likely to be great, given the lack of available treatments with liver-specific effects. NASH is form of nonalcoholic fatty liver disease (NAFLD) that can progress to cirrhosis, liver failure, and cancer. NASH is present in about 30 million adults in the US.

Intervention of Interest:

  • Obeticholic Acid (OCALIVA®, Intercept Pharmaceuticals, Inc.)

Date of Review: July 2016

For questions or additional information, please contact info@icer.org.

Final Documents

Below you will find the final documents from the policy paper review process:

NASH & PBC Documents:

PBC Documents Only:

NASH Documents Only: