Givosiran (Givlaari® )

Restricted Product(s)

• givosiran (Givlaari® ) subcutaneous injection for administration by a healthcare professional

FDA Approved Use

• For treatment of adults with acute hepatic porphyria (AHP)

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 18 years of age or older; AND
2. The patient has a confirmed diagnosis of acute hepatic porphyria (AHP) (including acute intermittent porphyria, hereditary coproporphyria, variegate porphyria, aminolevulinic acid (ALA) dehydratase deficient porphyria) [medical record documentation required]; AND
3. The patient has documentation of elevated urinary or plasma porphobilinogen (PBG) or ALA values [medical record documentation required]; AND
4. The patient has had one of the following [medical record documentation required]:
   1. History of at least two documented porphyria attacks within the 6 months prior to initiation of therapy with the requested agent (requiring hospitalization, urgent healthcare visit, or intravenous hemin administration at home); OR 
   2. History of one severe attack within the past year with central nervous system (CNS), autonomic nervous system (ANS), or peripheral nervous system (PNS) involvement (e.g., hallucinations, seizures, respiratory failure, paralysis); AND
5. The patient has not had and is not anticipating a liver transplantation [medical record documentation required]; AND 
6. The prescriber is a specialist in the area of the patient’s diagnosis (e.g., hepatologist, hematologist, neurologist) 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 [medical record documentation required]; AND 
3. The patient has had a positive clinical response while using the requested agent, as demonstrated by a reduction in porphyria attacks requiring hospitalization, urgent healthcare visit, or intravenous hemin administration [medical record documentation required]; AND 
4. The prescriber is a specialist in the area of the patient’s diagnosis (e.g., hepatologist, hematologist, neurologist) or has consulted with a specialist in the area of the patient’s diagnosis [medical record documentation required]; AND 
5. The requested quantity does NOT exceed the maximum units allowed for the duration of approval (see table below); AND 
6. 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)

*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. Stölzel U, Doss MO, Schuppan D, et al. Clinical guide and update on porphyrias. Gastroenterology. 2019;157(2):365-81. 
2. Kauppinen R. Porphyrias. Lancet. 2005;365:241-52.

Policy Implementation/Update Information

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