FDA Approves CTEXLI, the First-Ever Treatment for Cerebrotendinous Xanthomatosis, a Rare Lipid Storage Disorder
Mirum Pharmaceuticals, Inc. announced that the FDA has approved CTEXLI (chenodiol) tablets as the first and only treatment for cerebrotendinous xanthomatosis (CTX), a rare, progressive, and debilitating metabolic disease. CTX is caused by a deficiency of chenodeoxycholic acid (CDCA), leading to the toxic accumulation of bile alcohols and cholestanol, which contribute to severe neurological and systemic complications.
“The FDA’s approval of CTEXLI is tremendous as it unlocks an opportunity to better identify and treat adult patients with CTX in the Uni...
