Claims for Patent: 11,357,763

Summary for Patent: 11,357,763

Title:	Methods of treating fabry patients having renal impairment
Abstract:	Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in .alpha.-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.
Inventor(s):	Castelli; Jeff (New Hope, PA), Benjamin; Elfrida (Millstone Township, NJ)
Assignee:	Amicus Therapeutics, Inc. (Philadelphia, PA)
Application Number:	17/400,932
Patent Claims:	1. A method of treating Fabry disease in an ERT-experienced female patient with normal renal function with an eGFR of at least 90 mL/min/1.73 m.sup.2 and with a HEK assay amenable mutation in .alpha.-galactosidase A, the method comprising administering to the patient about 100 mg to about 150 mg free base equivalent (FBE) of migalastat at a frequency of once every other day. 2. The method of claim 1, wherein the administration of the migalastat is effective to stabilize renal function in the patient. 3. The method of claim 2, wherein administration of an effective amount of the migalastat to a group of ERT-experienced female patients having normal renal function with eGFRs of at least 90 mL/min/1.73 m.sup.2 and HEK assay amenable mutations in .alpha.-galactosidase A provides a mean annualized rate of change in eGFR.sub.CKD-EPI of greater than -1.5 mL/min/1.73 m.sup.2. 4. The method of claim 1, wherein the administration is effective to provide an annualized rate of change in eGFR.sub.CKD-EPI of greater than -3.0 mL/min/1.73 m.sup.2. 5. The method of claim 1, wherein the administration is effective to provide an annualized rate of change in eGFR.sub.CKD-EPI of greater than -1.5 mL/min/1.73 m.sup.2. 6. The method of claim 1, comprising administering about 123 mg FBE of migalastat at a frequency of every other day. 7. The method of claim 6, comprising administering about 150 mg of migalastat hydrochloride at a frequency of every other day. 8. The method of claim 6, wherein the migalastat is administered to the patient for at least 2 years. 9. The method of claim 8, wherein the migalastat is administered to the patient for at least 5 years. 10. The method of claim 6, wherein the migalastat is administered orally in solid dosage form. 11. A method of treating Fabry disease in an ERT-naive female patient with mild renal impairment with an eGFR of 60 to 90 mL/min/1.73 m.sup.2 and with a HEK assay amenable mutation in .alpha.-galactosidase A, the method comprising administering to the patient about 100 mg to about 150 mg free base equivalent (FBE) of migalastat at a frequency of once every other day. 12. The method of claim 11, wherein the administration of the migalastat is effective to stabilize renal function in the patient. 13. The method of claim 12, wherein administration of an effective amount of the migalastat to a group of ERT-experienced female patients having normal renal function with eGFRs of at least 90 mL/min/1.73 m.sup.2 and HEK assay amenable mutations in .alpha.-galactosidase A provides a mean annualized rate of change in eGFR.sub.CKD-EPI of greater than -0.1 mL/min/1.73 m.sup.2. 14. The method of claim 11, wherein the administration is effective to provide an annualized rate of change in eGFR.sub.CKD-EPI of greater than -4.0 mL/min/1.73 m.sup.2. 15. The method of claim 11, wherein the administration is effective to provide an annualized rate of change in eGFR.sub.CKD-EPI of greater than -0.1 mL/min/1.73 m.sup.2. 16. The method of claim 11, comprising administering about 123 mg FBE of migalastat at a frequency of every other day. 17. The method of claim 16, comprising administering about 150 mg of migalastat hydrochloride at a frequency of every other day. 18. The method of claim 16, wherein the migalastat is administered to the patient for at least 2 years. 19. The method of claim 18, wherein the migalastat is administered to the patient for at least 5 years. 20. The method of claim 16, wherein the migalastat is administered orally in solid dosage form.