Subject: Galafold 2650-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.
FDA-Approved Indication
Galafold is indicated for the treatment of adults with a confirmed
diagnosis of Fabry disease and an amenable galactosidase alpha
gene (GLA) variant based on in vitro assay data.
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. Initial requests: laboratory confirmation of an amenable
galactosidase alpha (GLA) gene variant.
B. Continuation requests: lab results or chart notes
documenting a positive response to therapy (e.g., reduction
in plasma globotriaosylceramide [GL-3, Gb3] or GL-3/Gb3
inclusions, improvement and/or stabilization in renal
function, pain reduction).
III. CRITERIA FOR INITIAL APPROVAL
Fabry disease with an amenable galactosidase alpha gene
(GLA) variant
Authorization of 12 months may be granted for treatment of
Fabry disease with an amenable galactosidase alpha gene (GLA)
variant when both of the following criteria are met:
A. Member has an amenable galactosidase alpha gene (GLA)
variant based on in vitro assay data; and
B. The requested medication will not be used in combination
with enzyme replacement therapy (ERT) for the treatment of
Fabry disease.
IV. CONTINUATION OF THERAPY
Authorization of 12 months may be granted for continued
treatment in members requesting reauthorization for an
indication listed in Section III who are responding to therapy
(e.g., reduction in plasma globotriaosylceramide [GL-3, Gb3] or
GL-3/Gb3 inclusions, improvement and/or stabilization in renal
function, pain reduction).
Place of Service:
Outpatient
The above policy is based on the following references:
- Galafold [package insert]. Philadelphia, PA: Amicus Therapeutics US, LLC; December 2022.
- Biegstraaten M, Arngrimsson R, Barbey F, et al. Recommendations for initiation and cessation of enzyme replacement therapy in patients with Fabry disease: the European Fabry Working Group consensus document. Orphanet J Rare Dis. 2015; 1036.
- Ortiz A, Germain DP, Desnick RJ, et al. Fabry disease revisited: Management and treatment recommendations for adult patients. Mol Genet Metab. 2018;123(4):416-427.
- Mehta A, Hughes DA. Fabry Disease. 2002 Aug 5 [Updated 2022 Jan 27]. In: Adam MP, Ardinger HH, Pagon RA, et al., editors. GeneReviews [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2022. Available from: https://www.ncbi.nlm.nih.gov/books/NBK1292/. Accessed May 18, 2022.
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.
August 18, 2023
