The U.S. Food and Drug Administration (FDA) has granted approval for Ctexli (chenodiol) as the first treatment for cerebrotendinous xanthomatosis (CTX), a rare genetic metabolic disorder.
Cerebrotendinous xanthomatosis is a condition that impairs the body’s ability to break down fats, leading to the accumulation of harmful cholesterol metabolites in various organs. This progressive buildup causes severe and often irreversible damage. Ctexli, the newly approved drug, aims to address this issue by replacing a missing bile acid, thereby reducing cholesterol deposits that contribute to the symptoms of CTX.
“The FDA remains committed to advancing treatment options for rare diseases, including those as uncommon as cerebrotendinous xanthomatosis,” said Dr. Janet Maynard, Director of the Office of Rare Diseases, Pediatrics, Urologic, and Reproductive Medicine at the FDA’s Center for Drug Evaluation and Research. “CTX is a multisystemic disorder that profoundly affects patients’ quality of life. This approval marks a significant milestone, offering a safe and effective treatment where none previously existed.”
Ctexli’s approval follows the results of a 24-week clinical trial, which demonstrated its ability to significantly reduce cholesterol metabolites in patients. The drug had previously been granted Priority Review, Fast Track, and Orphan Drug status by the FDA. The approval is a notable achievement for Mirum Pharmaceuticals Inc., the company behind the development of Ctexli.
With this landmark decision, patients suffering from CTX now have access to a treatment that may slow or prevent the progression of the disorder.
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