Novel Drug & Emergent Therapeutics | Pharma | Rare Diseases | Neurology
FDA Approves Ctexli: First Treatment for Cerebrotendinous Xanthomatosis
Time to read: 01:04
Published on MedED: 24 February 2025
Originally published: 21 February2025
Source: FDA Press Release
Type of article: Novel Drug & Emergent Therapy News
MedED Catalogue Reference: MNDR002
Category: Rare Diseases
Crossreference: Lipid Storage Disorders
Category tags: cerebrotendinous xanthomatosis (CTX), lipid storage disease
| Product Category | Product Name | Company | Status |
|---|---|---|---|
| FDA Approved |
The U.S. Food and Drug Administration (FDA) has approved Ctexli (chenodiol) as the first-ever treatment for cerebrotendinous xanthomatosis (CTX), a rare lipid storage disease that primarily affects adults. This approval represents a significant milestone in addressing a condition that previously lacked approved therapies, providing patients with a safe and effective management option. CTX is a progressive genetic metabolic disorder caused by a mutation in the CYP27A1 gene, leading to a deficiency in bile acid production and resulting in the accumulation of atypical cholesterol metabolites throughout the body, particularly in the brain, liver, skin, and tendons.
Ctexli works by replenishing the deficient bile acids, thereby reducing these harmful cholesterol deposits and alleviating associated clinical abnormalities. The efficacy of Ctexli was demonstrated in a rigorous 24-week double-blind, placebo-controlled trial, which showed significant reductions in plasma cholestanol and urine 23S-pentol levels in patients receiving the treatment.
However, the prescribing information for Ctexli includes a warning about potential liver toxicity, especially in patients with pre-existing liver conditions. Patients are advised to undergo regular liver function tests and to consult their healthcare provider if they experience any signs of liver damage. Overall, Ctexli's approval is a crucial development for those affected by CTX.
Access the Press Release
24 February 2025 | BusinessWire | Mirum’s CTEXLI™ (chenodiol) Tablets Receives FDA Approval for Treatment of Cerebrotendinous Xanthomatosis (CTX)
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