Drug intelligence / Profile preview

obeticholic acid

Development stage
Approved
Lead developer
Alfasigma
Modality
Orthosteric Ligands → Classical Binding Small Molecules → Small Molecules
Administration
Oral
01

Overview

Obeticholic acid is a semi-synthetic bile acid analogue and a potent agonist of the farnesoid X receptor (FXR), which plays a critical role in regulating bile acid, lipid, and glucose metabolism. It is used to treat primary biliary cholangitis (PBC), an autoimmune liver disease that leads to progressive destruction of the bile ducts, fibrosis, and cirrhosis. Obeticholic acid can be used alone in patients intolerant to ursodeoxycholic acid (UDCA) or in combination with UDCA for those who have had an inadequate response. By activating FXR in liver and intestinal cells, obeticholic acid reduces hepatic synthesis of bile acids, increases their secretion from the liver (choleresis), suppresses inflammation and fibrosis, and helps limit cholestatic injury[1][2][3][5][6][8]. The drug was granted accelerated approval by the FDA in 2016 under the brand name Ocaliva for PBC; however, its full approval status has been under review as of late 2024[5].

Brand names
Ocaliva
Other names
6α-ethyl-chenodeoxycholic acid
02

Targets

FXR (Farnesoid x-activated receptor)

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