Restricted Product(s):

• lumasiran (Oxlumo^®) subcutaneous injection for administration by a healthcare professional 

FDA Approved Use:

• For treatment of adult and pediatric patients (birth and older) with primary hyperoxaluria type 1 (PH1) to lower urinary and plasma oxalate levels

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 has a diagnosis of primary hyperoxaluria type 1 (PH1) [medical record documentation required]; AND
2. The diagnosis has been confirmed by at least one of the following [medical record documentation required]:
   1. Molecular genetic testing demonstrating AGXT gene mutation; OR
   2. Liver biopsy demonstrating alanine-glyoxylate aminotransferase (AGT) deficiency; AND
3. The requested agent will be used to lower urinary and/or plasma oxalate levels [medical record documentation required]; AND
4. The patient has NOT had a previous liver transplant [medical record documentation required]; AND
5. The patient does NOT have any FDA labeled contraindications to the requested agent [medical record documentation required]; AND
6. The prescriber is a specialist in the area of the patient’s diagnosis (e.g., nephrologist, gastroenterologist, urologist, geneticist) or has consulted with a specialist in the area of the patient’s diagnosis [medical record documentation required]; AND
7. The requested quantity does NOT exceed the maximum units allowed for the duration of approval (see table below); AND
8. 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 demonstrated a positive clinical response while using the medication, as demonstrated by improvement, stabilization, or slowed worsening of disease [e.g., reduction from baseline in laboratory parameters (e.g., 24-hour urinary oxalate excretion, spot urinary oxalate:creatinine ratio, plasma oxalate concentration), improvement/stabilization/slowed worsening of clinical manifestations (e.g., eGFR, nephrocalcinosis, renal stone events, systemic oxalosis, renal impairment)] [medical record documentation required]; AND
4. The patient has NOT had a previous liver transplant [medical record documentation required]; AND
5. The patient does NOT have any FDA labeled contraindications to the requested agent [medical record documentation required]; AND
6. The prescriber is a specialist in the area of the patient’s diagnosis (e.g., nephrologist, gastroenterologist, urologist, geneticist) or has consulted with a specialist in the area of the patient’s diagnosis [medical record documentation required]; AND
7. The requested quantity does NOT exceed the maximum units allowed for the duration of approval (see table below); AND
8. 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

*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.

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.

March 2025: Criteria change: Removed requirement that the patient has an eGFR greater than or equal to 30 mL/minute/1.73 m². Updated maximum units for clarity per FDA label.

November 2021: Criteria change: Removed baseline diagnostic metabolic testing requirements, and no secondary causes of hyperoxaluria; added use to lower urinary oxalate levels and no FDA labeled contraindications to therapy for clarity according to FDA labeling; reformatted continuation criteria to require a positive clinical response while using the medication as demonstrated by improvement, stabilization, or slowed worsening of disease.

October 2021: Criteria change: Added Site of Care medical necessity criteria. Policy notification given 8/2/2021 for effective date 10/1/2021.

July 2021: Criteria update: Added HCPCS code J0224 to dosing reference table effective 7/1/2021, deleted C9074 and non-specific codes C9399, J3490, and J3590 termed 6/30/2021.

June 2021: Criteria change: Added maximum units; medical policy formatting change. Policy notification given 4/16/2021 for effective date 6/16/2021.

*Further historical criteria changes and updates available upon request from Medical Policy and/or Corporate Pharmacy.