Restricted Product(s):

• pozelimab-bbfg (Veopoz^™) intravenous infusion or subcutaneous injection for administration by a healthcare professional

FDA Approved Use:

• For treatment of adult and pediatric patients 1 year of age and older with CD55-deficient protein-losing enteropathy (PLE), also known as CHAPLE disease

Criteria for Medical Necessity:

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

Initial Criteria for Approval:

1. The patient is 1 year of age or older; AND

2. The patient has a diagnosis of CD55-deficient protein-losing enteropathy (PLE)/CHAPLE disease [medical record documentation required]; AND

3. The patient has a confirmed biallelic CD55 loss-of-function mutation detected by genotype analysis [medical record documentation required]; AND

4. The patient has active disease, defined as BOTH of the following:

    a. Hypoalbuminemia (i.e., serum albumin concentration of less than or equal to 3.2 g/dL) [medical record documentation required];

AND

    b. Presence of one or more of the following signs or symptoms within the last 6 months [medical record documentation required]:

        i. Abdominal pain; OR

        ii. Diarrhea; OR

        iii. Peripheral edema; OR

        iv. Facial edema; AND

5. The patient has received meningococcal vaccination prior to starting therapy with the requested agent [medical record documentation required]; AND

    a. If risk of delaying treatment with the requested agent outweighs risk of meningococcal infection, the patient will receive antibacterial drug prophylaxis for meningococcal infection [medical record documentation required]; AND

6. The patient does NOT have presence of any concomitant disease leading to and/or other apparent cause of hypoproteinemia (e.g., protein malnutrition, urinary protein loss, impaired protein synthesis due to liver disease) [medical record documentation required]; AND

7. The patient does NOT have presence of any concomitant disease that leads to secondary intestinal lymphangiectasia (e.g., a fontan procedure for congenital heart disease) [medical record documentation required]; AND

8. The patient will NOT be using the requested agent in combination with any other complement inhibitors (e.g., eculizumab [Soliris^®]) [medical record documentation required]; AND

9. The prescriber is a specialist in the area of the patient’s diagnosis (e.g., gastroenterologist, hematologist, immunologist) or has consulted with a specialist in the area of the patient’s diagnosis [medical record documentation required]; AND

10. The requested quantity does NOT exceed the maximum units allowed for the duration of approval (see table below); AND

11. 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:

180 days (6 months)

Continuation Criteria for Approval:

1. The patient was approved through Blue Cross NC initial criteria for approval; OR

2. The patient would have met initial criteria for approval at the time they started therapy; AND

3. The patient has had clinically significant improvement or disease stabilization while receiving treatment with the requested agent (i.e., normalization of serum albumin levels; improvement of abdominal pain, diarrhea, and/or edema) [medical record documentation required]; AND

4. The patient will NOT be using the requested agent in combination with any other complement inhibitors (e.g., eculizumab [Soliris ^®]) [medical record documentation required]; AND

5. The prescriber is a specialist in the area of the patient’s diagnosis (e.g., gastroenterologist, hematologist, immunologist) 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:

365 days (1 year)

*Maximum units allowed for duration of approval
**Non-specific assigned HCPCS codes, must submit requested product NDC

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:

    a. History of mild adverse events that have not been successfully managed through mild pre-medication (e.g., diphenhydramine, acetaminophen, steroids, fluids, etc.); OR

    b. Inability to physically and cognitively adhere to the treatment schedule and regimen complexity; OR

    c. New to therapy, defined as initial injection or infusion OR less than 3 months since initial injection or infusion; OR

    d. Re-initiation of therapy, defined as ONE of the following:

        i. 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

        ii. 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

    e. 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.

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.

April 2024: Coding change: Added HCPCS code J9376 to dosing reference table effective 4/1/2024; deleted C9399, J3490, J3590 termed 3/31/2024.

November 2023: Original medical policy criteria issued.