Ctexli offers hope for patients with a previously untreated metabolic disorder, Cerebrotendinous Xanthomatosis.
The U.S. Food and Drug Administration (FDA) has approved Ctexli (chenodiol) as the first treatment for cerebrotendinous xanthomatosis (CTX), a rare genetic disorder affecting the body’s ability to break down fats. CTX leads to harmful cholesterol metabolite buildup in various organs, causing progressive damage. Ctexli works by replacing a missing bile acid, reducing cholesterol deposits linked to CTX symptoms.
“The FDA is dedicated to supporting new drug development for rare diseases including very rare metabolic diseases like cerebrotendinous xanthomatosis,” said Director Janet Maynard of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, in the FDA’s Center for Drug Evaluation and Research. “CTX is a progressive multisystemic disorder that significantly impacts patients and previously lacked approved treatments. Today’s approval provides a safe and effective treatment option for CTX.”
The drug’s effectiveness was shown in a 24-week clinical trial, where it significantly reduced cholesterol metabolites in patients. The FDA granted Ctexli Priority Review, Fast Track, and Orphan Drug status, with approval going to Mirum Pharmaceuticals Inc.
As the Lord Leads, Pray with Us…
- For Director Maynard as she oversees the Office of Rare Diseases, Pediatrics, Urologic, and Reproductive Medicine.
- For Acting Commissioner Sarah Brenner as she manages the Food and Drug Administration.
Sources: Food and Drug Administration
