Subject: Galafold 2650-A SGM P2021

Drug

GALAFOLD  (migalastat)

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: alpha- galactosidase enzyme assay or genetic testing results supporting diagnosis. In the case of obligate carriers, the documentation must be submitted for the parent.
        B.  Continuation requests: chart notes documenting a positive response to therapy (e.g., reduction in plasma globotriaosylceramide [GL-3] or GL-3 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 all of the following criteria are met:
        A.  The diagnosis of Fabry disease was confirmed by enzyme assay demonstrating a deficiency
             of alpha-galactosidase enzyme activity or by genetic testing, or the member is a
             symptomatic obligate carrier; and 
        B.  Member has an amenable galactosidase alpha gene (GLA) variant based on in vitro assay
             data; and
        C.  Galafold 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 Fabry disease with an amenable galactosidase alpha gene (GLA) variant
        who are responding to therapy (e.g., reduction in plasma globotriaosylceramide [GL-3] or GL-3 inclusions, improvement and/or stabilization in renal function, pain reduction).

 

Place of Service:

Outpatient

The above policy is based on the following references:

1. Galafold [package insert]. Cranbury, NJ: Amicus Therapeutics U.S., Inc.; September 2020.
2. 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.
3. 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.

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.

September 30, 2021