Pharmacy Prior Authorization Criteria

Galafold (migalastat)

For diagnosis of Fabry Disease
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.

Prior Authorization (Initial Authorization)

Length of Approval: 12 Month(s)

AND

Detection of pathogenic mutations in the GLA gene by molecular genetic testing
Deficiency in α-galactosidase A (α-Gal A) enzyme activity in plasma, isolated leukocytes, or dried blood spots (DBS)
Significant clinical manifestations (e.g., neuropathic pain, cardiomyopathy, renal insufficiency, angiokeratomas, cornea verticillata)

AND

Patient has an amenable galactosidase alpha gene (GLA) variant based on in vitro assay data [A]

AND

Prior Authorization (Reauthorization)

Length of Approval: 24 Month(s)

Patient demonstrates positive clinical response to therapy as evidenced by one of the following: [3, 4]

Reduction in plasma or urinary sediment lyso-GL-3, GL-3 compared to baseline
Reduction in number of GL-3 inclusions per kidney interstitial capillary (KIC) in renal biopsy samples compared to baseline
Improvement and/or stabilization in symptoms (e.g., renal function, neuropathic pain)

AND

2024-09-04, 2023-11-01, 2023-08-21, 2022-09-30, 2021-10-29, 2021-10-21, 2020-10-02, 2019-09-19

Galafold prescribing information. Amicus Therapeutics U.S., Inc. Cranbury, NJ. June 2024.
Per clinical consultation with geneticist. October 11, 2018.
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. doi:10.1016/j.ymgme.2018.02.014.
Michaud M, Mauhin W, Belmatoug N, et al. When and How to Diagnose Fabry Disease in Clinical Pratice. Am J Med Sci. 2020;360(6):641-649. doi:10.1016/j.amjms.2020.07.011.

2024-09-04: 2024 Annual Review. Updated language in reauthorization criteria. Background updates.
2023-11-01: Annual Review, no changes.
2023-08-21: update guideline
2022-09-30: 2022 UM Annual Review.
2021-10-29: Update to include OptumRx SP - EHB Formulary
2021-10-21: Background updates.
2020-10-02: Annual review, removed drug name from criteria
2019-09-19: Annual Review - guideline updated