Subject: Ultomiris 2835-A SGM P2023a
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 Indications
1. Ultomiris is indicated for the treatment of adult and pediatric
patients one month of age and older with paroxysmal
nocturnal hemoglobinuria (PNH).
2. Ultomiris is indicated for the treatment of adults and
pediatric patients one month of age and older with atypical
hemolytic uremic syndrome (aHUS) to inhibit complement-
mediated thrombotic microangiopathy (TMA).
3. Ultomiris is indicated for the treatment of adult patients with
generalized myasthenia gravis (gMG) who are anti-
acetylcholine receptor (AChR) antibody-positive.
Limitations of Use: Ultomiris 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. Paroxysmal nocturnal hemoglobinuria: flow cytometry
used to show results of glycosylphosphatidylinositol
anchored proteins (GPI-APs) deficiency
2. Atypical hemolytic uremic syndrome: ADAMTS 13 level
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
B. For continuation requests: Chart notes or medical record
documentation supporting positive clinical response.
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) 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
B. Atypical hemolytic uremic syndrome (aHUS)
Authorization of 6 months may be granted for treatment of
atypical hemolytic uremic syndrome (aHUS) not caused by
Shiga toxin when all of the following criteria are met:
1. Absence of Shiga toxin
2. ADAMTS 13 activity level above 5%
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
IV. CONTINUATION OF THERAPY
A. 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).
B. Atypical hemolytic uremic syndrome (aHUS)
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).
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 member demonstrates a
positive response to therapy (e.g., improvement in MG-ADL
score, changes compared to baseline in Quantitative
Myasthenia Gravis (QMG) total score).
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:
- Ultomiris [package insert]. Boston, MA: Alexion Pharmaceuticals, Inc.; July 2022.
- Parker CJ. Management of paroxysmal nocturnal hemoglobinuria in the era of complement inhibitory therapy. 2011; 21-29.
- Lee JW, Sicre de Fontbrune F, Wong LL, et al. Ravulizumab (ALXN1210) vs eculizumab in adult patients with PNH naive to complement inhibitors: The 301 study. Blood. 2018 Dec 3; pii: blood-2018-09-876136.
- 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.
- 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. Update on the diagnosis and management of paroxysmal nocturnal hemoglobinuria. Hematology Am Soc Hematol Educ Program. 2016;2016(1):208-216.
- Sanders D, Wolfe G, Benatar M et al. International consensus guidance for management of myasthenia gravis. Neurology. 2021; 96 (3) 114-122.
- Tuan Vu, Andreas Meisel, Renato Mantegazza, et al. Terminal Complement Inhibitor Ravulizumab in Generalized Myasthenia Gravis. NEJM Evid 2022; 1 (5)
- 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.
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
