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US 2021/0069162 A1Published application
Methods Of Treating Fabry Patients Having Renal Impairment
AssigneeAmicus Therapeutics, Inc.
InventorsJeff Castelli, Elfrida Benjamin
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Abstract
Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in α-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.
Claims20
A method of stabilizing renal function in a patient diagnosed with Fabry disease and having renal impairment, the method comprising:
- administering to a Fabry disease patient about 100 mg to about 150 mg free base equivalent (FBE) of migalastat at a frequency of once every other day, wherein the Fabry disease patient has moderate or severe renal impairment with an eGFR less than 60 mL/min/1.73 m2 and a HEK assay amenable α-galactosidase A mutation,
- wherein the administration is effective to (i) reduce left ventricular mass index (LVMi) in the patient, and (ii) provide a mean annualized rate of change in eGFRCKD-EPI of greater than −1.0 mL/min/1.73 m2.
Dependencies are inferred from the claim text.