FDA Approves First Treatment for Rare Genetic Disorder CTX

The U.S. Food and Drug Administration (FDA) has approved Ctexli (chenodiol), the first treatment for cerebrotendinous xanthomatosis (CTX), a rare genetic metabolic disorder affecting adults. CTX, caused by a mutation in the CYP27A1 gene, leads to abnormal cholesterol breakdown, causing harmful deposits in organs like the brain, liver, and tendons. Ctexli replaces deficient bile acids, reducing these deposits and addressing clinical abnormalities.  

The approval follows a 24-week trial showing Ctexli significantly reduced cholesterol metabolites in patients. However, the drug carries a warning for liver toxicity, requiring regular liver tests. Common side effects include diarrhea, headache, and abdominal pain.  

Dr. Janet Maynard of the FDA emphasized the importance of this milestone for CTX patients, who previously had no approved treatments. Ctexli, developed by Mirum Pharmaceuticals Inc., received Priority Review, Fast Track, and Orphan Drug designations. The recommended dosage is 250 mg, taken three times daily.

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