Claims for Patent: 11,278,537

Summary for Patent: 11,278,537

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:	17/400,626
Patent Claims:	1. A method of stabilizing renal function in a Fabry patient with moderate or severe renal impairment with an eGFR less than 60 mL/min/1.73 m.sup.2 and with a HEK assay amenable .alpha.-galactosidase A mutation, 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, wherein the administration is effective to (i) increase the mean white blood cell (WBC) .alpha.-galactosidase A activity in the patient, and (ii) provide a mean annualized rate of change in eGFR.sub.CKD-EPI of greater than -1.0 mL/min/1.73 m.sup.2. 2. The method of claim 1, wherein the patient has an eGFR of 30 to 59 mL/min/1.73 m.sup.2. 3. The method of claim 1, wherein the patient has an eGFR of 35 to 59 mL/min/1.73 m.sup.2. 4. The method of claim 1, wherein the patient has an eGFR of less than 30 mL/min/1.73 m.sup.2. 5. The method of claim 1, wherein the patient has an eGFR of 15 to 29 mL/min/1.73 m.sup.2. 6. The method of claim 1, wherein the patient has a proteinuria level of less than 100 mg/24 hr prior to initiating the administration of the migalastat. 7. The method of claim 1, wherein the patient has a proteinuria level of 100 to 1,000 mg/24 hr prior to initiating the administration of the migalastat. 8. The method of claim 1, wherein the patient has a proteinuria level of greater than 1,000 mg/24 hr prior to initiating the administration of the migalastat. 9. The method of claim 1, wherein the migalastat is in a solid dosage form. 10. The method of claim 1, wherein the migalastat is administered orally. 11. The method of claim 1, wherein the migalastat is administered for at least 28 days. 12. The method of claim 1, wherein the migalastat is administered for at least 6 months. 13. The method of claim 1, wherein the migalastat is administered for at least 12 months. 14. The method of claim 1, comprising administering about 123 mg FBE of migalastat. 15. The method of claim 1, comprising administering about 123 mg of migalastat free base. 16. The method of claim 1, comprising administering about 150 mg of migalastat hydrochloride. 17. The method of claim 1, wherein the patient has an eGFR of 30 to 59 mL/min/1.73 m.sup.2, and wherein the administration is effective to provide a mean increase in WBC .alpha.-galactosidase A activity of about 1.4 4 MU/hr/mg. 18. The method of claim 1, wherein the HEK assay amenable mutation is a mutation that, when the mutation is expressed in HEK-293 cells incubated in the presence of 10 .mu.M migalastat compared to HEK-293 cells without migalastat, is shown to have (1) a relative increase of at least 20% .alpha.-galactosidase A activity and (2) an absolute increase of at least 3% of the wild-type .alpha.-galactosidase A activity. 19. The method of claim 1, wherein the migalastat is administered as a pharmaceutically acceptable salt. 20. The method of claim 1, wherein the patient is an enzyme replacement therapy (ERT)-naive patient.