Subject: Soliris 3537-A SGM P2023a
Policy:
I. INDICATIONS
The indications below including FDA-approved indications and
compendial uses are considered covered benefits provided that
all the approval criteria are met and the member has no
exclusions to the prescribed therapy.
FDA-Approved Indications
A. Paroxysmal nocturnal hemoglobinuria (PNH) to reduce
hemolysis
B. Atypical hemolytic uremic syndrome (aHUS) to inhibit
complement-mediated thrombotic microangiopathy
C. Generalized myasthenia gravis (gMG) in adult patients who
are anti-acetylcholine receptor (AchR) antibody positive
D. Neuromyelitis optica spectrum disorder (NMOSD) in adult
patients who are anti-aquaporin-4 (AQP4) antibody positive
Limitations of Use: Soliris is not indicated for the treatment of
patients with Shiga toxin E. coli related hemolytic uremic
syndrome (STEC-HUS).
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 for new requests for treatment of:
A. For initial requests:
1. Atypical hemolytic uremic syndrome: ADAMTS 13 level
2. Paroxysmal nocturnal hemoglobinuria: flow cytometry
used to show results of glycosylphosphatidylinositol-
anchored proteins (GPI-APs) deficiency
3. Generalized myasthenia gravis: anti-acetylcholine
receptor (AchR) antibody positive, clinical classification of
myasthenia gravis score, MG activities of daily living
score, use of IVIG, use of two immunosuppressive
therapies
4. Neuromyelitis optica spectrum disorder: immunoassay
used to confirm anti-aquaporin-4 (AQP4) antibody is
present
B. For continuation requests: Chart notes or medical record
documentation supporting positive clinical response.
III. CRITERIA FOR INITIAL APPROVAL
A. Atypical hemolytic uremic syndrome
Authorization of 6 months may be granted for treatment of
atypical hemolytic uremic syndrome not caused by Shiga
toxin when all of the following criteria are met:
1. ADAMTS 13 activity level above 5%
2. Absence of Shiga toxin
B. 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) as demonstrated by either of the
following:
a. At least 5% PNH cells
b. At least 51% of GPI-AP deficient poly-morphonuclear
cells
2. Flow cytometry is used to demonstrate GPI-APs deficiency
C. Generalized myasthenia gravis (gMG)
Authorization of 6 months may be granted for treatment of
generalized myasthenia gravis (gMG) when all of the
following criteria are met:
1. Anti-acetylcholine receptor (AchR) antibody positive
2. Myasthenia Gravis Foundation of America (MGFA) clinical
classification II to IV
3. MG activities of daily living (MG-ADL) total score ≥6
4. Meets both of the following:
a. Member has had an inadequate response to at least
two immunosuppressive therapies listed below:
i. azathioprine
ii. cyclosporine
iii. mycophenolate mofetil
iv. tacrolimus
v. methotrexate
vi. cyclophosphamide
vii. rituximab
b. Member has inadequate response to chronic IVIG
D. Neuromyelitis Optica Spectrum Disorder (NMOSD)
Authorization of 6 months may be granted for treatment of
neuromyelitis optica spectrum disorder (NMOSD) when all of
the following criteria are met:
1. Anti-aquaporin-4 (AQP4) antibody positive
2. Member exhibits one of the following core clinical
characteristics of NMOSD:
a. Optic neuritis
b. Acute myelitis
c. Area postrema syndrome (episode of otherwise
unexplained hiccups or nausea and vomiting)
d. Acute brainstem syndrome
e. Symptomatic narcolepsy or acute diencephalic clinical
syndrome with NMOSD-typical diencephalic MRI
lesions
f. Symptomatic cerebral syndrome with NMOSD-typical
brain lesions
3. The member will not receive the requested drug
concomitantly with other biologics for the treatment of
NMOSD.
IV. CONTINUATION OF THERAPY
A. Atypical hemolytic uremic syndrome
Authorization of 12 months may be granted for continued
treatment in members requesting reauthorization when there
is no evidence of unacceptable toxicity or disease progression
while on the current regimen and demonstrate a positive
response to therapy (e.g., normalization of lactate
dehydrogenase (LDH) levels, platelet counts).
B. Paroxysmal nocturnal hemoglobinuria
Authorization of 12 months may be granted for continued
treatment in members requesting reauthorization when there
is no evidence of unacceptable toxicity or disease progression
while on the current regimen and demonstrate a positive
response to therapy (e.g., improvement in hemoglobin
levels, normalization of lactate dehydrogenase [LDH] levels).
C. Generalized myasthenia gravis (gMG)
Authorization of 12 months may be granted for continued
treatment in members requesting reauthorization when there
is no evidence of unacceptable toxicity or disease progression
while on the current regimen and demonstrate a positive
response to therapy (e.g., improvement in MG-ADL score,
changes compared to baseline in Quantitative Myasthenia
Gravis (QMG) total score).
D. Neuromyelitis optica spectrum disorder (NMOSD)
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., reduction in number of relapses).
3. The member will not receive the requested drug
concomitantly with other biologics for the treatment of
NMOSD.
V. DOSAGE AND ADMINISTRATION
Approvals may be subject to dosing limits in accordance with
FDA-approved labeling, accepted compendia, and/or evidence-
based practice guidelines.
Place of Service:
Outpatient
The above policy is based on the following references:
- Soliris [package insert]. New Haven, CT: Alexion Pharmaceuticals, Inc.; November 2020.
- Loirat C, Fakhouri F, Ariceta G, et al. An international consensus approach to the management of atypical hemolytic uremic syndrome in children. Pediatr Nephrol. Published online: April 11, 2015.
- Parker CJ. Management of paroxysmal nocturnal hemoglobinuria in the era of complement inhibitory therapy. 2011; 21-29.
- Sanders D, Wolfe G, Benatar M et al. International consensus guidance for management of myasthenia gravis. Neurology. 2021; 96 (3) 114-122 .
- Jaretzki A, Barohn RJ, Ernstoff RM et al. Myasthenia Gravis: Recommendations for Clinical Research Standards. Ann Thorac Surg. 2000;70: 327-34.
- Hillmen P, Young NS, Schubert J, et al. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. 2006;335:1233-43.
- Howard JF, Utsugisawa K, Benatar M. Safety and efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory generalized myasthenia gravis (REGAIN); a phase 3, randomized, double-blind, placebo-controlled, multicenter study. Lancet Neurol. 2017 Oct 20. http://dx.doi.org/10.1016/S1474-4422(17)30369-1Ingenix HCPCS Level II, Expert 2011.
- Brodsky RA, Young NS, Antonioli E, et al. Multicenter phase 3 study of the complement inhibitor eculizumab for the treatment of patients with paroxysmal nocturnal hemoglobinuria. Blood. 2008;111(4):1840-1847.
- 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.
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 31, 2023
