Niemann-Pick Disease- NC Standard

Restricted Product(s)

Restriction applies to brand and generic products.

• Miplyffa™ (arimoclomol) 
• Aqnuersa™ (levacetylleucine)

FDA Approved Use

• Miplyffa 
  • For use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older. 
• Aqnuersa 
  • For the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg.

Criteria for Approval of Restricted Product(s)

Initial Coverage Criteria

1. The patient has a confirmed diagnosis of Niemann-Pick disease Type C (NPC) (medical record documentation required); AND
2. The genetic analysis (deoxyribonucleic acid [DNA] sequence) confirms mutations in NPC1 or NPC2 genes (medical record documentation required); AND
3. The request is for the following: 
   1. Miplyffa: 
      1. The patient is 2 years of age or older (medical record documentation required); AND 
      2. The patient will be using the requested medication in combination with miglustat for the treatment of neurological manifestations (medical record documentation required); OR 
   2. Aqnuersa: 
      1. The patient weighs ≥15 kg (medical record documentation required); AND
4. The patient has documented symptoms related to NPC’s neurological manifestations (i.e., gait, sitting, stance, speech disturbance and fine motor skills) (medical record documentation required); AND 
5. The patient is being managed by or in consultation with a specialist in the area of the patient’s diagnosis (e.g., endocrinologist, geneticist); AND 
6. The prescribed dose and quantity are appropriate based on intended use and FDA labeling; AND 
7. For formularies that exclude (non-formulary) the requested medication, Non-formulary Exception Criteria applies. 

Duration of Approval: 365 days (1 year)

Continuation Coverage Criteria

1. The patient has been previously approved with Blue Cross and Blue Shield of North Carolina (Blue Cross NC) or would have met initial criteria for approval upon the start of therapy; AND 
2. The patient has experienced clinical benefit since initiation of therapy as indicated by improvement in neurological manifestations (i.e., gait, sitting, stance, speech disturbance and fine motor skills) (medical record documentation required); AND 
3. The patient continues to be managed by or in consultation with a specialist in the area of the patient’s diagnosis (e.g., endocrinologist, geneticist); AND 
4. The prescribed dose and quantity are appropriate based on intended use and FDA labeling.

Duration of Approval: 365 days (1 year)

Quantity Limit Exception Criteria

1. The quantity (dose) requested is for documented titration purposes at the initiation of therapy (authorization for a 90 day titration period); AND 
2. The prescribed dose cannot be achieved using a lesser quantity of a higher strength; AND 
3. The quantity (dose) requested does not exceed the maximum FDA labeled dose, when specified, or to the safest studied dose per the manufacturer’s product insert; OR 
4. If the quantity (dose) requested exceeds the maximum FDA labeled dose, when specified, or to the safest studied dose per the manufacturer’s product insert, then the prescriber must submit documentation in support of therapy with a higher dose for the intended diagnosis (submitted documentation may include medical records OR fax form which reflects medical record documentation that shows the length of time the requested dose has been used, and what other medications and doses have been tried and failed). 

Duration of Approval: 365 days (1 year)

References

All information referenced is from FDA package insert unless otherwise noted below.

Policy Implementation/Update Information

Criteria and treatment protocols are reviewed annually by the Blue Cross NC P&T Committee, regardless of change. This policy is reviewed in Q1 annually.

November 2024: Criteria change: Updated policy to remove requirement of filipin-staining and biomarkers as diagnostic as it is no longer considered a method of diagnosis. Updated grammatical error in policy. 

October 2024: Original utilization management criteria issued.