- Ctexli (chenodiol) approved for treating cerebrotendinous xanthomatosis (CTX) in adults.
- Reduces plasma cholestanol and urine 23S-pentol levels.
- CTX is caused by mutations in the CYP27A1 gene, leading to metabolic dysfunction.
- Liver toxicity warnings and common adverse events included in prescribing information.
- Recommended dosing regimen is 250 mg orally three times daily.
FDA Approves First Cerebrotendinous Xanthomatosis Treatment
Conexiant
February 21, 2025
