Ctexli™

Drug - Ctexli™ (chenodiol) [Mirum Pharmaceuticals, Inc.]

January 2026

Therapeutic Area - Bile Salts

Initial approval criteria

  • The patient has a diagnosis of cerebrotendinous xanthomatosis (CTX) as confirmed by ONE of the following:
    • Genetic testing confirming variants in the CYP27A1 gene OR
    • ALL of the following:
      • Elevated plasma cholestanol greater than or equalto 5 to 10 times ULN (upper limit of normal) OR
      • Urine positive for bile alcohols AND
      • Clinical findings consistent for CTX [e.g., xanthomas (present in lungs, tendons, bone or central nervous system), infantile-onset diarrhea, childhood-onset cataract(s), adult-onset progressive neurologic dysfunction (dementia, psychiatric disturbances, pyramidal and/or cerebellar signs, dystonia, atypical parkinsonism, peripheral neuropathy, and seizures]

          AND

  • The patient’s age is within FDA labeling for the requested indication AND
  • The patient has had a baseline liver transaminase (alanine aminotransferase [ALT] and aspartate aminotransferase [AST]) and total bilirubin level prior to initiating the requested agent AND
  • Ctexli is prescribed by or in consultation with a specialist in the area of the patient’s diagnosis AND
  • The patient does NOT have any FDA labeled contraindications to the requested agent

Renewal criteria

  • The patient has had clinical benefit with the requested agent AND
  • The patient is monitored for changes in liver transaminase (alanine aminotransferase [ALT] and aspartate aminotransferase [AST]) and total bilirubin level AND BOTH of the following:
    • The patient has liver transaminase levels less than 3 times the upper limit of normal (ULN) AND
    • The patient has a total bilirubin level less than 2 times the ULN AND
  • Ctexli is prescribed by or in consultation with a specialist in the area of the patient’s diagnosis AND
  • The patient does NOT have any FDA labeled contraindications to the requested agent

Quantity limits

  • 102 tablets per 34 days

Questions

Provider Call Center: (844) 575-7887