Obeticholic acid, sold under the brand name Ocaliva, is a semi-synthetic bile acid analogue which has the chemical structure 6α-ethyl-chenodeoxycholic acid. It is used as a medication used to treat primary biliary cholangitis. Chemically it is C26H44O4 with a molecular weight near 420.6 g/mol and appears as a white to off-white crystalline powder that is practically insoluble in water but soluble in common organic solvents. Pharmacologically it is a potent farnesoid X receptor FXR agonist that downregulates bile acid synthesis via suppression of CYP7A1, modulates bile acid transporters, and exerts anti-inflammatory and antifibrotic effects relevant to cholestatic liver disease. Typical API quality control employs HPLC, LC-MS and NMR for identity and purity, and formulations are manufactured to control particle size, polymorphic form and residual solvents to meet regulatory standards.
Parent: Obeticholic Acid
Parent: Obeticholic Acid
Parent: Obeticholic Acid
Parent: Obeticholic Acid
Parent: Obeticholic Acid
Parent: Obeticholic Acid
Parent: Obeticholic Acid
Parent: Obeticholic Acid
Parent: Obeticholic Acid
Parent: Obeticholic Acid
Parent: Obeticholic Acid
Parent: Obeticholic Acid
Typical specification targets for obeticholic acid API include assay acceptance near 98.0 to 102.0 percent on an anhydrous basis and related substances controlled by validated chromatographic methods; commonly applied limits are individual unidentified impurities at or below 0.5 percent and total impurities at or below 1.0 percent, with tighter limits for specified known degradants or epimers often set at 0.1 to 0.2 percent. Known related compounds and potential impurities include stereoisomeric epimers at the 6 position, 3-keto and dehydro oxidation products, acetylated or esterified impurities and low-level process-related residuals; each of these is typically assigned a specification limit and monitored by HPLC-UV or LC-MS, while heavy metals, residual solvents and microbial limits are controlled to ICH and pharmacopeial thresholds.
Obeticholic acid is indicated for the treatment of primary biliary cholangitis PBC, typically as an adjunct to ursodeoxycholic acid UDCA in patients with an inadequate response or as monotherapy for those who are intolerant to UDCA; its therapeutic effects stem from FXR activation leading to reduced bile acid synthesis, improved cholestasis biomarkers and potential antifibrotic activity.
Obeticholic acid has been studied in nonalcoholic fatty liver disease NAFLD and nonalcoholic steatohepatitis NASH and has shown some improvement in fibrosis in clinical trials, but it is not broadly approved for fatty liver disease and its use is investigational in this setting; potential benefits must be weighed against adverse effects such as pruritus and changes in lipid profile and current clinical guidance does not support routine use for simple fatty liver.
Obeticholic acid and ursodeoxycholic acid UDCA have different mechanisms of action; OCA is a selective FXR agonist that reduces bile acid synthesis and modulates metabolic and fibrotic pathways, whereas UDCA is a hydrophilic bile acid that improves cholestasis primarily by replacing toxic bile acids and enhancing bile flow; OCA is used when patients have an insufficient biochemical response to UDCA because it can produce additional reductions in alkaline phosphatase and other cholestatic markers, but OCA is not universally superior and choice depends on individual response and safety profile.
Yes, obeticholic acid was approved by the U.S. Food and Drug Administration for the treatment of primary biliary cholangitis under the brand name Ocaliva; the approval includes specific dosing, monitoring requirements and warnings for patients with advanced liver disease, and prescribers should follow current labeling and safety communications.
