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Drug - Galafold™ (migalastat) [Amicus Therapeutics]

June 2019

Therapeutic area - Fabry Disease

Initial approval criteria

  • Patient is ≥ 18 years old AND
  • Patient has a documented diagnosis of Fabry disease with biochemical/genetic confirmation by 1 of the following:
    • Males only: α-galactosidase A (α-Gal A) activity in plasma, isolated leukocytes, and/or cultured cells OR
    • Plasma or urinary globotriaosylceramide(Gb3/GL-3) or globotriaosylsphingosine (lyso-Gb3) OR
    • Detection of pathogenic mutations in the GALA/GLA gene by molecular genetic testing AND
  • Patient has an amenable GLA mutation (as defined in the migalastat labeling) determined by or in consult with a clinical genetics professional as causing Fabry disease (pathogenic) AND
  • If taking an angiotensin-converting enzyme inhibitors (ACE-I) or angiotensin II receptor blocker (ARB), patient must be stable on therapy for at least 4 weeks AND
  • Baseline echocardiogram, estimate glomerular filtration rate (eGFR), 24-hour urine protein, urine GL-3 and/or GL-3 inclusions, and alpha-galactosidase (α-Gal, male patients only) must be performed prior to initiation AND
  • Patient has not undergone or scheduled to undergo kidney transplantation or currently on dialysis AND
  • Will NOT be used in combination with agalsidase beta
  • Initial approval is for 6 months

Renewal criteria

  • Patient continues to meet initial criteria AND
  • Disease response with treatment as defined by a reduction in urine GL-3 and/or GL-3 inclusions compared to pre-treatment baseline AND
  • Absence of unacceptable toxicity (e.g., kidney infections) and absence of progression into renal impairment or end-stage renal disease (e.g., eGFR < 30 mL/min/1.73 m2)
  • Renewal approval is for 6 months

Quantity limits

  • 14 capules every 28 days


MHCP Provider Call Center 651-431-2700 or 800-366-5411

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