Ocaliva

Ocaliva is the brand name for obeticholic acid, an oral medication developed by Intercept Pharmaceuticals. It is a semisynthetic bile acid derivative that acts as a farnesoid X receptor (FXR) agonist, and it modulates bile acid synthesis, inflammation, and fibrosis in the liver.

Indication and approval: It is approved for adults with primary biliary cholangitis (PBC) who have an inadequate response to or are intolerant of ursodeoxycholic acid (UDCA).

Mechanism and use: By activating FXR, obeticholic acid lowers bile acid synthesis and can improve cholestasis. In PBC, it can improve biochemical markers and may slow disease progression when added to UDCA. It is taken as a once-daily oral tablet, with a typical starting dose of 5 mg, which can be increased to 10 mg daily based on tolerance and response.

Safety: Common adverse effects include pruritus, fatigue, and gastrointestinal symptoms. Liver function tests should be monitored; there is a potential risk of liver injury, particularly in patients with advanced liver disease, and the drug is used with caution in such patients.

Research: Ocaliva has been investigated for nonalcoholic steatohepatitis (NASH) and related fibrosis, but it is not approved for this indication.

History and status: Obeticholic acid was developed by Intercept and received regulatory approval in 2016 for PBC in the United States and in other regions. It represents a pharmacologic approach targeting FXR in cholestatic liver disease.