Subject: Galafold 2650-A SGM P2024

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.

          This indication is approved under accelerated approval based on reduction in kidney interstitial
          capillary cell globotriaosylceramide (KIC GL-3) substrate. Continued approval for this indication
          may be contingent upon verification and description of clinical benefit in confirmatory trials.

          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) variant.
          B.  Continuation requests: lab results or chart notes documenting a positive response to
               therapy.


   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:

1. Galafold [package insert]. Philadelphia, PA: Amicus Therapeutics US, LLC; June 2023.
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.
4. Mehta A, Hughes DA. Fabry Disease. 2002 Aug 5 [Updated 2023 Mar 9]. In: Adam MP, Feldman J, Mirzaa GM, et al., editors. GeneReviews [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2024. Available from: https://www.ncbi.nlm.nih.gov/books/NBK1292/. Accessed February 2, 2024.

 

 

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, 2024