Subject: Cerezyme 2051-A SGM P2021

Drug

CEREZYME  (imiglucerase)

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 long-term enzyme replacement therapy (ERT) for pediatric and adult patients
      with a confirmed diagnosis of type 1 Gaucher disease that results in one or more of the following conditions:
      anemia, thrombocytopenia, bone disease, 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 in members requesting
reauthorization for Gaucher disease type 1, type 2, or type 3 who are 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:

1. Cerezyme [package insert]. Cambridge, MA: Genzyme Corporation; April 2018.
2. 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.
3. 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.
4. Pastores GM, Hughes DA. Gaucher Disease. [Updated February 26, 2015]. In: Pagon RA, Adam MP, Ardinger HH, et al, editors. GeneReviews® [Internet]. Seattle, WA: University of Washington, Seattle; 1993-2016.
5. 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.
6. American Society of Health System Pharmacists. AHFS Drug Information. Bethesda, MD. Electronic version, 2021. Available with subscription. URL: http://online.lexi.com/crlsql/servlet/crlonline. Accessed January 28, 2021.
7. DRUGDEX System (electronic version). Micromedex Truven Health Analytics. Available with subscription. URL: www.micromedexsolutions.com. Accessed January 28, 2021.

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.

May 26, 2021