chenodiol — Blue Cross Blue Shield of New Mexico

cerebrotendinous xanthomatosis (CTX)

Initial criteria

ONE of the following:
A. BOTH of the following:
1. ONE of the following:
A. The patient has a diagnosis of cerebrotendinous xanthomatosis (CTX) as confirmed by ONE of the following:
1. Genetic testing confirming variants in the CYP27A1 gene OR
2. ALL of the following:
A. Elevated plasma cholestanol ≥ 5 to 10 times ULN AND
B. Urine positive for bile alcohols AND
C. Clinical findings consistent for CTX (e.g., xanthomas, infantile-onset diarrhea, childhood-onset cataracts, adult-onset progressive neurologic dysfunction)
B. The patient has another FDA labeled indication for the requested agent and route of administration
2. If the patient has an FDA labeled indication, then ONE of the following:
A. The patient’s age is within FDA labeling for the indication OR
B. There is support for using the agent for the patient’s age
3. The patient has had a baseline liver transaminase (ALT and AST) and total bilirubin level prior to initiating the requested agent
4. The prescriber is a specialist in the area of the patient’s diagnosis (e.g., gastroenterologist, geneticist, hepatologist, endocrinologist, neurologist) or has consulted with a specialist
5. The patient does NOT have any FDA labeled contraindications to the requested agent

1. The patient has been previously approved for the requested agent through the plan’s Prior Authorization process
2. The patient has had clinical benefit with the requested agent
3. The patient is monitored for changes in liver transaminase (ALT and AST) and total bilirubin level AND BOTH of the following:
A. Liver transaminase levels < 3× ULN AND
B. Total bilirubin < 2× ULN
4. The prescriber is a specialist in the area of the patient’s diagnosis or has consulted with a specialist
5. The patient does NOT have any FDA labeled contraindications to the requested agent

12 months