Galafold™

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

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

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