The US FDA has approved chenodiol for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. It is the first FDA-approved drug for CTX, a very rare lipid storage disease. Chenodiol is a bile acid that may compensate for deficient levels of endogenous chenodeoxycholic acid in CTX patients, helping to reduce the abnormal accumulation of cholesterol metabolites.
Clinical evidence In a 24-week clinical trial, chenodiol (250 mg three times per day) significantly reduced cholesterol metabolites, such as plasma cholestanol and urine 23S-pentol, which are markedly increased in CTX patients, compared to placebo treatment. Dosage and administration Available as tablets Before initiating chenodiol, obtain baseline liver transaminase (alanine aminotransferase [ALT] and aspartate aminotransferase [AST]) and total bilirubin levels in all patients 250 mg orally three times daily…
