arimoclomol citrate — Blue Cross Blue Shield of Illinois

Niemann-Pick disease Type C

Initial criteria

1. ONE of the following:
A. The requested agent is eligible for continuation of therapy AND the following:
- The prescriber states the patient has been treated with the requested agent (not samples) within the past 90 days AND is at risk if therapy is changed OR
B. ALL of the following:
1. The patient has a diagnosis of Niemann-Pick disease Type C AND
2. Genetic analysis confirms mutation in the NPC1 or NPC2 genes AND
3. The patient has disease-related neurological symptoms AND
4. The requested agent will be used in combination with miglustat AND
5. If the patient has an FDA labeled indication, ONE of the following:
A. The patient’s age is within FDA labeling for the requested indication OR
B. There is support for using the requested agent for the patient’s age for the requested indication AND
2. The prescriber is a specialist in the area of the patient’s diagnosis (e.g., geneticist) or has consulted with a specialist AND
3. 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 AND
2. The patient has had clinical benefit with the requested agent AND
3. The prescriber is a specialist in the area of the patient’s diagnosis (e.g., geneticist) or has consulted with a specialist AND
4. The patient does NOT have any FDA labeled contraindications to the requested agent

12 months