This program allows physicians to request permission from Amicus Therapeutics (Amicus) for treatment access to migalastat hydrochloride (HCl) for specific adult patients with Fabry disease. Treatment is open label for 6 months with renewal every 6 months.
This Physician Initiated Request program allows physicians to request permission from Amicus
to receive migalastat HCl for specific patients with Fabry disease who have a mutation
amenable to this treatment, who do not have access to available treatment alternatives, or do
not meet requirements for participation in an existing migalastat clinical study. Up to 20
patients worldwide may be treated. Patients must meet specific criteria to receive Amicus
permission for participation. Key criteria for participation include: 16-74 years old;
Confirmed GLA gene mutation shown to be responsive to migalastat; Have no treatment option
because either unsuitable for enzyme replacement therapy (ERT) or unable to access ERT.
Requirements for sufficient kidney function. If permission is granted, initial approval is
for a 6 month supply of migalastat HCl with renewal every 6 months available upon meeting
continued eligibility.
Drug: migalastat HCl
150 mg capsule taken every other day by mouth. An inactive reminder capsule may be provided to take on the days in between migalastat HCl
Other Name: AT1001, migalastat
Inclusion Criteria:
- Confirmed GLA mutation predicted to be responsive migalastat in the human embryonic
kidney (HEK-293) cell-based assay
- 16-74 years of age
- Strong clinical indication for treatment of Fabry disease
- No other treatment option including either unsuitable for ERT or unable to access ERT
- Appropriate female and male contraception
- Willing to receive treatment with migalastat HCl via this program including having
signed an authorization for sharing clinical data
Exclusion Criteria:
- Estimated glomerular filtration rate (eGFR) or GFR <30 mL/minute
- Scheduled for renal or other organ transplant or replacement therapy
- Receiving GLYSET® (miglitol), ZAVESCA® (miglustat) or enzyme replacement therapy
FABRAZYME® (agalsidase beta) or REPLAGAL™ (agalsidase alpha)
- Contraindication to migalastat, i.e., sensitivity to other iminosugar such as
miglustat, miglitol
- Treated with another investigational drug within 30 days of start of migalastat HCl
treatment
- Unable to comply with study requirements or deemed otherwise unsuitable for study
entry in the opinion of the investigator.
CHOC Children's Hospital Division of Metabolic Disease
Orange, California, United States
University of Maryland Medical Center
Baltimore, Maryland, United States
Kidney Care and Transplant Services of New England
Springfield, Massachusetts, United States
Icahn School of Medicine at Mount Sinai
New York, New York, United States
Royal Melbourne Hospital
Parkville, Victoria, Australia
Medical Monitor, Clinical Research, Study Director
Amicus Therapeutics