Ctexli FDA Approval History
Last updated by Judith Stewart, BPharm on Feb 24, 2025.
FDA Approved: Yes (First approved February 21, 2025)
Brand name: Ctexli
Generic name: chenodiol
Dosage form: Tablets
Company: Mirum Pharmaceuticals Inc.
Treatment for: Cerebrotendinous Xanthomatosis
Ctexli (chenodiol) is a synthetic form of the bile acid chenodeoxycholic acid indicated for the treatment of cerebrotendinous xanthomatosis in adults.
- Cerebrotendinous xanthomatosis (CTX) is a rare, genetic bile acid synthesis disorder caused by a mutation in the CYP27A1 gene resulting in a deficiency of sterol 27-hydroxylase, a mitochondrial enzyme involved in converting cholesterol into chenodeoxycholic acid. CTX is characterized by the accumulation of deposits of cholesterol and cholestanol the nerve cells and membranes potentially causing damage to the brain, spinal cord, tendons, lens of the eye and arteries.
- Ctexli works by replacing deficient levels of chenodeoxycholic acid to reduce abnormal deposits of the cholesterol metabolites thought to be responsible for clinical abnormalities in CTX.
- Chenodiol was first approved under the brand name Chenix in 1983 and reapproved as Chenodal in 2009 for the treatment of gallstones. Ctexli is the first treatment containing chenodiol that is specifically indicated to treat CTX.
- FDA approval for Ctexli was supported by data from a randomized, double-blind, placebo controlled, crossover trial (NCT 04270682) which showed that patients treated with Ctexli demonstrated a significant reduction in plasma cholesterol metabolites (cholestanol and urine 23S-pentol) compared to patients treated with placebo.
- Ctexli tablets are administered orally three times daily.
- Warnings and precautions associated with Ctexli include hepatotoxicity. Baseline liver transaminase (alanine aminotransferase [ALT] and aspartate aminotransferase [AST]) and total bilirubin levels should be obtained prior to initiating treatment with Ctexli.
- Common adverse reactions (incidence > 14%) include diarrhea, headache, abdominal pain, constipation, hypertension, muscular weakness, and upper respiratory tract infection.
Development timeline for Ctexli
| Date | Article |
|---|---|
| Feb 24, 2025 | Approval FDA Approves Ctexli (chenodiol) for Cerebrotendinous Xanthomatosis |
Further information
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