Empaveli – NC Standard

Restricted Product(s)

• EmpaveliT® (pegcetacoplan)

FDA Approved Use

• For the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH)

Criteria for Approval of Restricted Product(s)

Initial Criteria for Approval

1. The patient is 18 years of age or older; AND 
2. The patient has a confirmed diagnosis of paroxysmal nocturnal hemoglobinuria (PNH) (medical record documentation required); AND 
3. ONE of the following: 
   1. The patient has a hemoglobin ≤ 7 g/dL (medical record documentation required); OR 
   2. The patient has hemoglobin ≤ 9 g/dL and is experiencing symptoms of anemia (medical record documentation required); OR 
   3. The patient has documented symptoms of thromboembolic complications (abdominal pain, shortness of breath, chest pain, end organ damage) (medical record documentation required); OR 
   4. The patient is transitioning from another complement inhibitor approved for the diagnosis of PNH (e.g. eculizumab or ravulizumab); AND 
      1. The patient was previously approved through Blue Cross NC initial criteria for eculizumab or ravulizumab; AND 
4. The patient will NOT be using the requested agent in combination with another complement inhibitor used to treat PNH (e.g., eculizumab, ravulizumab); OR 
   1. The patient is transitioning from eculizumab to pegcetacoplan; AND 
   2. The patient will discontinue eculizumab 4 weeks after initiation of pegcetacoplan therapy; AND 
5. The prescriber is a specialist in the area of the patient’s diagnosis (e.g., hematologist, oncologist, or immunology specialist) or the prescriber has consulted with a specialist in the area of the patient’s diagnosis; AND 
6. For formularies that exclude (non-formulary) the requested medication, Non-formulary Exception Criteria applies.
   
   Duration of Approval: 180 days (6 months)

Continuation Criteria for Approval

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 (medical record documentation required); AND 
2. The patient has experienced stabilization or improvement of symptoms from baseline while using the requested agent, as demonstrated by the following (medical record documentation required): 
   1. Significant reduction in transfusion requirements (medical record documentation required); AND 
   2. No thromboembolism events persisting while using the requested agent (medical record documentation required); AND 
3. The patient will NOT be using the requested agent in combination with another complement inhibitor used to treat PNH (e.g. eculizumab, ravulizumab) (medical record documentation required).
   
   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

June 2023: Criteria update: Criteria review and formatting changes. Added duration of approval to quantity limit exception criteria.
June 2021: Original utilization management policy issued.