Subject: Fabhalta 6288-A SGM P2024b
Policy:
I. INDICATIONS
The indications below including FDA-approved indications and compendial uses are
considered a covered benefit provided that all the approval criteria are met and the member
has no exclusions to the prescribed therapy.
FDA-Approved Indication
Fabhalta is indicated for:
A. Treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH).
B. To reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of
rapid disease progression, generally a urine protein-to-creatinine ratio (UPCR) ≥1.5 g/g*.
*This indication is approved under accelerated approval based on reduction of proteinuria. It
has not been established whether Fabhalta slows kidney function decline in patients with IgAN.
Continued approval for this indication may be contingent upon verification and description of
clinical benefit in a confirmatory clinical trial.
All other indications are considered experimental/investigational and not medically necessary.
II. DOCUMENTATION
Submission of the following information is necessary to initiate the prior authorization review:
A. For initial requests:
1. Paroxysmal nocturnal hemoglobinuria: Flow cytometry used to show results of
glycosylphosphatidylinositol-anchored proteins (GPI-APs) deficiency.
2. Primary immunoglobin A nephropathy (IgAN):
i. Kidney biopsy confirming a diagnosis of primary immunoglobulin A nephropathy
(IgAN).
ii. Laboratory report and/or chart note(s) indicating the member has proteinuria greater
than or equal to 1 g/day or baseline UPCR greater than or equal to 0.8 g/g based on
a 24-hour urine collection.
B. For continuation requests:
1. Paroxysmal nocturnal hemoglobinuria: Chart notes or medical record documentation
supporting positive clinical response.
2. Primary immunoglobin A nephropathy (IgAN): Laboratory report and/or chart note(s)
indicating the member has decreased levels of proteinuria or UPCR from baseline based
on a 24-hour urine collection.
III. CRITERIA FOR INITIAL APPROVAL
A. Paroxysmal nocturnal hemoglobinuria
Authorization of 6 months may be granted for treatment of paroxysmal nocturnal
hemoglobinuria (PNH) when all of the following criteria are met:
1. The diagnosis of PNH was confirmed by detecting a deficiency of
glycosylphosphatidylinositol-anchored proteins (GPI-APs) (e.g., at least 5% PNH cells, at
least 51% of GPI-AP deficient poly-morphonuclear cells).
2. Flow cytometry is used to demonstrate GPI-APs deficiency.
3. Member has and exhibits clinical manifestations of disease (e.g., LDH > 1.5 ULN,
thrombosis, renal dysfunction, pulmonary hypertension, dysphagia).
4. The requested medication will not be used in combination with another complement
inhibitor (e.g., Empaveli, Piasky, Soliris, Ultomiris) for the treatment of PNH.
B. Primary immunoglobulin A nephropathy (IgAN)
Authorization of 10 months may be granted for treatment primary immunoglobulin A
nephropathy (IgAN) when all of the following criteria are met:
1. Member has a diagnosis of primary immunoglobulin A nephropathy (IgAN) confirmed by
kidney biopsy.
2. Member has proteinuria greater than or equal to 1 g/day or UPCR greater than or equal
to 0.8 g/g based on a 24-hour urine collection.
3. Member has received a stable dose of maximally tolerated renin-angiotensin system
(RAS) inhibitor therapy (e.g., angiotensin converting enzyme inhibitor [ACEI] or
angiotensin II receptor blocker [ARB]) for at least 3 months prior to initiation of therapy,
or member has an intolerance or contraindication to RAS inhibitors.
4. Member has experienced an intolerance to oral glucocorticoid (e.g., prednisone).
IV. CONTINUATION OF THERAPY
A. Paroxysmal nocturnal hemoglobinuria
Authorization of 12 months may be granted for continued treatment in members requesting
reauthorization when all of the following criteria are met:
1. There is no evidence of unacceptable toxicity or disease progression while on the
current regimen.
2. The member demonstrates a positive response to therapy (e.g., improvement in
hemoglobin levels, normalization of lactate dehydrogenase [LDH] levels).
3. The requested medication will not be used in combination with another complement
inhibitor (e.g., Empaveli, Piasky, Soliris, Ultomiris) for the treatment of PNH.
B. Primary immunoglobulin A nephropathy (IgAN)
Authorization of 12 months may be granted for continued treatment in members requesting
reauthorization when all of the following criteria are met:
1. There is no evidence of unacceptable toxicity or disease progression while on the
current regimen.
2. The member is experiencing benefit from therapy as evidenced by either of the following:
i. Decreased levels of proteinuria from baseline on a 24-hour urine collection.
ii. Decrease in UPCR from baseline based on a 24-hour urine collection.
Place of Service:
Outpatient
The above policy is based on the following references:
- Fabhalta [package insert]. East Hanover, NJ: Novartis Pharmaceuticals Corporation; August 2024.
- Parker CJ. Management of paroxysmal nocturnal hemoglobinuria in the era of complement inhibitory therapy. 2011; 21-29.
- Borowitz MJ, Craig F, DiGiuseppe JA, et al. Guidelines for the Diagnosis and Monitoring of Paroxysmal Nocturnal Hemoglobinuria and Related Disorders by Flow Cytometry. Cytometry B Clin Cytom. 2010: 78: 211-230.
- Preis M, Lowrey CH. Laboratory tests for paroxysmal nocturnal hemoglobinuria (PNH). Am J Hematol. 2014;89(3):339-341.
- Parker CJ. Update on the diagnosis and management of paroxysmal nocturnal hemoglobinuria. Hematology Am Soc Hematol Educ Program. 2016;2016(1):208-216.
- Dezern AE, Borowitz MJ. ICCS/ESCCA consensus guidelines to detect GPI-deficient cells in paroxysmal nocturnal hemoglobinuria (PNH) and related disorders part 1 - clinical utility. Cytometry B Clin Cytom. 2018 Jan;94(1):16-22.
- Kidney Disease: Improving Global Outcomes (KDIGO) Glomerular Diseases Work Group. KDIGO 2021 Clinical Practice Guideline for the Management of Glomerular Diseases. Kidney Int. 2021 Oct; 100 (4S): S1-S276. doi: 10.1016/j.kint.2021.05.021.
Copyright Aetna Inc. All rights reserved. Pharmacy Clinical Policy Bulletins are developed by Aetna to assist in administering plan benefits and constitute neither offers of coverage nor medical advice. This Clinical Policy Bulletin contains only a partial, general description of plan or program benefits and does not constitute a contract. Aetna does not provide health care services and, therefore, cannot guarantee any results or outcomes. Participating providers are independent contractors in private practice and are neither employees nor agents of Aetna or its affiliates. Treating providers are solely responsible for medical advice and treatment of members. This Clinical Policy Bulletin may be updated and therefore is subject to change.
December 11, 2024
