Subject: Cerezyme 2051-A SGM P2023
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.
A. FDA-Approved Indications
Cerezyme is indicated for treatment of adults and pediatric
patients 2 years of age and older with Type 1 Gaucher
disease that results in one or more of the following
conditions: anemia, thrombocytopenia, bone disease, and/or
hepatomegaly or splenomegaly.
B. Compendial Uses
1. Gaucher disease type 2
2. Gaucher disease type 3
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: beta-glucocerebrosidase enzyme
assay or genetic testing results supporting diagnosis.
III. CRITERIA FOR INITIAL APPROVAL
A. Gaucher disease type 1
Authorization of 12 months may be granted for treatment of
Gaucher disease type 1 when the diagnosis of Gaucher
disease was confirmed by enzyme assay demonstrating a
deficiency of beta-glucocerebrosidase (glucosidase) enzyme
activity or by genetic testing.
B. Gaucher disease type 2
Authorization of 12 months may be granted for treatment of
Gaucher disease type 2 when the diagnosis of Gaucher
disease was confirmed by enzyme assay demonstrating a
deficiency of beta-glucocerebrosidase (glucosidase) enzyme
activity or by genetic testing.
C. Gaucher disease type 3
Authorization of 12 months may be granted for treatment of
Gaucher disease type 3 when the diagnosis of Gaucher
disease was confirmed by enzyme assay demonstrating a
deficiency of beta-glucocerebrosidase (glucosidase) enzyme
activity or by genetic testing.
IV. CONTINUATION OF THERAPY
Authorization of 12 months may be granted for continued
treatment of an indication listed in Section III when all of the
following criteria are met:
A. Member meets the criteria for initial approval.
B. Member is not experiencing an inadequate response or any
intolerable adverse events from therapy.
Place of Service:
Outpatient
The above policy is based on the following references:
- Cerezyme [package insert]. Cambridge, MA: Genzyme Corporation; December 2021.
- Altarescu G, Hill S, Wiggs E, et al. The efficacy of enzyme replacement therapy in patients with chronic neuronopathic Gaucher's disease. J Pediatr. 2001;138:539-547.
- Erikson A, Forsberg H, Nilsson M, Astrom M, Mansson JE. Ten years’ experience of enzyme infusion therapy of Norrbottnian (type 3) Gaucher disease. Acta Paediatr. 2006;95:312-317.
- Pastores GM, Hughes DA. Gaucher Disease. 2000 July 27 [Updated June 21, 2018]. In: Adam MP, Everman DB, Mirzaa GM, et al, editors. GeneReviews® [Internet]. Seattle, WA: University of Washington, Seattle; 1993-2022.
- Kaplan P, Baris H, De Meirleir L, et al. Revised recommendations for the management of Gaucher disease in children. Eur J Pediatr. 2013;172:447-458.
- National Organization for Rare Disorders. (2003). NORD guide to rare disorders. Philadelphia: Lippincott Williams & Wilkins.
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.
June 18, 2023
