• ADAMTS13, recombinant-krhn (Adzynma^® ) intravenous infusion for administration by a healthcare professional 

• For prophylactic or on demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP) 

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

1. The patient is 2 years of age or older; AND
2. he patient has a diagnosis of congenital thrombotic thrombocytopenic purpura (cTTP) / severe hereditary ADAMTS13 deficiency [medical record documentation required]; AND
3. The patient’s diagnosis has been confirmed by BOTH of the following:
   1. Molecular genetic testing demonstrating biallelic pathogenic variants in ADAMTS13 [medical record documentation required]; AND
   2. ONE of the following:
      1. Severe ADAMTS13 deficiency defined by ADAMTS13 activity less than 10%, as measured by fluorescent resonance energy transfer-von Willebrand factor 73 (FRETS-VWF73) assay [medical record documentation required]; OR
      2. If the patient is currently receiving standard of care prophylactic therapy (e.g., plasma infusion), then ADAMTS13 activity may exceed 10% [medical record documentation required]; AND
4. he patient does NOT have a medical history nor presence of functional ADAMTS13 inhibitors prior to initiating therapy with the requested agent [medical record documentation required]; AND
5. The patient has NOT been diagnosed with any other TTP-like disorders (e.g., macroangiopathic hemolytic anemia, acquired TTP, Evans Syndrome) [medical record documentation required]; AND
6. If the request if for prophylactic therapy, ONE of the following:
   1. The patient has a history of at least ONE documented TTP event [medical record documentation required]; OR
   2. The patient is currently receiving prophylactic therapy (e.g., plasma infusion) [medical record documentation required]; AND
7. If the request is for on demand therapy, the patient is at risk of a disease exacerbation [medical record documentation required]; AND
8. The prescriber is a specialist in the area of the patient’s diagnosis (e.g., hematologist, immunologist, intensive care specialist, specialist in rare genetic hematologic diseases) or has consulted with a specialist in the area of the patient’s diagnosis [medical record documentation required]; AND 
9. The requested quantity does NOT exceed the maximum units allowed for the duration of approval (see table below) [medical record documentation required]; AND
10. For requests for injection or infusion administration of the requested medication in an inpatient or outpatient hospital setting, Site of Care Criteria applies.

Duration of Approval: 180 days (6 months)

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 demonstrated a positive clinical response while using the requested agent (e.g., improvement in acute and subacute TTP events, platelet counts, microangiopathic hemolytic anemia episodes, strokes/transient ischemic attacks, other clinical symptoms) [medical record documentation required]; AND
4. The patient has NOT been diagnosed with any other TTP-like disorders (e.g., macroangiopathic hemolytic anemia, acquired TTP, Evans Syndrome) [medical record documentation required]; AND
5. The prescriber is a specialist in the area of the patient’s diagnosis (e.g., hematologist, immunologist, intensive care specialist, specialist in rare genetic hematologic diseases) 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) [medical record documentation required]; 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.

Duration of Approval: 365 days (1 year)

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.

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

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.

July 2024: Coding change: Added HCPCS code J7171 to dosing reference table effective 7/1/2024; deleted C9167, J3490, and J3590 termed 6/30/2024.

April 2024: Coding change: Added HCPCS code C9167 to dosing reference table effective 4/1/2024; deleted C9399 termed 3/31/2024. Adjusted maximum units according to coding unit definition for clarity.

February 2024: Original medical policy criteria issued.