Pegcetacoplan (Empaveli™)

Restricted Product(s)

• pegcetacoplan (Empaveli™) subcutaneous infusion for administration by a healthcare professional

FDA Approved Use

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

Criteria for Medical Necessity

The restricted product(s) may be considered medically necessary when the following criteria are met:

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 a hemoglobin ≤ 9 g/dL and the patient is experiencing symptoms of anemia [medical record documentation required]; OR 
   3. The patient has documented symptoms of thromboembolic complications (e.g., 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 treatment of PNH (e.g., eculizumab or ravulizumab) [medical record documentation required]; AND
      1. The patient was previously approved through Blue Cross NC initial criteria for approval for eculizumab or ravulizumab [medical record documentation required]; OR 
      2. The patient would have met Blue Cross NC initial criteria for approval for eculizumab or ravulizumab at the time they started therapy with these agents [medical record documentation required]; AND 
4. The patient will NOT be using the requested agent in combination with another complement inhibitor used to treat PNH (e.g., crovalimab, eculizumab, iptacopan, ravulizumab) [medical record documentation required]; OR 
   1. The patient is transitioning from eculizumab to pegcetacoplan therapy [medical record documentation required]; AND 
   2. The patient will discontinue eculizumab 4 weeks after initiation of pegcetacoplan therapy [medical record documentation required]; AND 
5. The prescriber is a specialist in the area of the patient’s diagnosis (e.g., hematologist, oncologist, immunology specialist) or has consulted with a specialist in the area of the patient’s diagnosis [medical record documentation required]; AND 
6. The requested quantity does NOT exceed the maximum units allowed for the duration of approval (see table below); AND
7. For requests for injection or infusion administration of the requested medication in an inpatient or outpatient hospital setting, Site of Care Criteria applies (outlined below)*
   
   Duration of Approval: 90 days (one-time initial approval)
   
   *** ***NOTE: This medical policy applies to initial coverage requests only. For continuation of coverage, please submit requests using the member’s pharmacy benefit prior authorization submission process. For additional information, please visit Blue Cross

*Site of Care Medical Necessity Criteria

1. For requests for injection or infusion administration in an inpatient setting, the injection or infusion may be given if the above medical necessity criteria are met AND the inpatient admission is NOT for the sole purpose of administering the injection or infusion; OR 
2. For requests for injection or infusion administration in an outpatient hospital setting, the injection or infusion may be given if the above medical necessity criteria are met AND ONE of the following must be met: 
   1. History of mild adverse events that have not been successfully managed through mild pre-medication (e.g., diphenhydramine, acetaminophen, steroids, fluids, etc.); OR 
   2. Inability to physically and cognitively adhere to the treatment schedule and regimen complexity; OR 
   3. New to therapy, defined as initial injection or infusion OR less than 3 months since initial injection or infusion; OR 
   4. Re-initiation of therapy, defined as ONE of the following: 
      1. First injection or infusion after 6 months of no injections or infusions for drugs with an approved dosing interval less than 6 months duration; OR
      2. First injection or infusion after at least a 1-month gap in therapy outside of the approved dosing interval for drugs requiring every 6 months dosing duration; OR 
   5. Requirement of a change in the requested restricted product formulation; AND 
3. If the Site of Care Medical Necessity Criteria in #1 or #2 above are not met, the injection or infusion will be administered in a home-based infusion or physician office setting with or without supervision by a certified healthcare professional. 

References

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

1. Hillmen P, Szer J, Weitz I, et al. Pegcetacoplan versus eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2021 Mar;384(11):1028-1037.

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 Q2 annually.

September 2024: Criteria update: Updated list of complement inhibitors not to be used concomitantly to add newly approved crovalimab for clarity.
January 2024: Criteria update: Updated list of complement inhibitors not to be used concomitantly for clarity.
August 2021: Original medical policy criteria issued.