The US Food and Drug Administration has approved a small molecule drug to treat a rare genetic disorder that can cause progressive damage to the brain and other areas of the body. The drug, Ctexli (chenodiol), is a bile acid replacement therapy developed to treat cerebrotendinous xanthomatosis (CTX), a lipid storage disorder. Patients with CTX are deficient in an enzyme that helps the body break down fats.The disorder is caused by a reduced production of bile acid in the liver, resulting in the deposit of atypical cholesterol metabolites in the brain, liver and other parts of the body.
