Subject: miglustat-Zavesca 2098-A SGM P2022
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ZAVESCA
(miglustat)
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miglustat
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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
Zavesca is indicated as monotherapy for the treatment of
adult patients with mild to moderate type 1 Gaucher
disease for whom enzyme replacement therapy is not a
therapeutic option (e.g. due to allergy, hypersensitivity, or
poor venous access).
B. Compendial Uses
Niemann-Pick disease, type C
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. Gaucher disease type 1: beta-glucocerebrosidase enzyme
assay or genetic testing results supporting diagnosis
B. Niemann-Pick disease, type C: genetic testing results
showing mutations in NPC1 or NPC2
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 ALL of the following criteria
are met:
1. The diagnosis of Gaucher disease was confirmed by
enzyme assay demonstrating a deficiency of beta-
glucocerebrosidase (glucosidase) enzyme activity or by
genetic testing, and
2. The member has a documented inadequate response to,
intolerable adverse events with, or a clinical reason to
not use enzyme replacement therapy (e.g., allergy,
hypersensitivity, poor venous access).
B. Niemann-Pick disease, type C
Authorization of 12 months may be granted for treatment of
Niemann-Pick disease, type C when the diagnosis was
confirmed by genetic testing results showing mutations in
NPC1 or NPC2 genes.
IV. CONTINUATION OF THERAPY
A. Gaucher disease type 1
Authorization of 12 months may be granted for continued
treatment in members requesting reauthorization for
Gaucher disease type 1 who are not experiencing an
inadequate response or any intolerable adverse events from
therapy.
B. Niemann-Pick disease, type C
Authorization of 12 months may be granted for continued
treatment in members requesting reauthorization for
Niemann-Pick disease, type C 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:
- Zavesca [package insert]. South San Francisco, CA: Actelion Pharmaceuticals US, Inc.; January 2021.
- miglustat [package insert]. Horsham, PA: Patriot Pharmaceuticals, LLC.; January 2021.
- Lexicomp Online, Lexi-Drugs Online. Waltham, MA: UpToDate, Inc.; April 2, 2021. https://online.lexi.com. Accessed November 12, 2021.
- DRUGDEX System (electronic version). Micromedex Truven Health Analytics. Available with subscription. URL: micromedexsolutions.com. Accessed Novemeber 12, 2021.
- 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 26, 2022
