Claims for Patent: 10,874,655

Summary for Patent: 10,874,655

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 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.
Inventor(s):	Jeff Castelli, Elfrida Benjamin
Assignee:	Bpcr LP , Amicus Therapeutics Inc
Application Number:	US16/678,183
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for patent 10,874,655
Patent Claims:	1. A method of treating Fabry disease in a human patient having moderate renal impairment with an eGFR of 30 to 59 mL/min/1.73 m2, the method comprising administering to the patient a therapeutically effective dose of migalastat at a frequency of once every other day, wherein the therapeutically effective dose is about 100 mg to about 150 mg free base equivalent (FBE), wherein the patient has a HEK assay amenable mutation in α-galactosidase A, and wherein the dose is not adjusted relative to a patient without renal impairment. 2. The method of claim 1, wherein the patient is an enzyme replacement therapy (ERT)-experienced patient. 3. The method of claim 1, wherein the patient is an enzyme replacement therapy (ERT)-naïve patient. 4. 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. 5. 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. 6. 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. 7. The method of claim 1, wherein the migalastat is administered orally. 8. The method of claim 7, wherein the migalastat is in a solid dosage form. 9. The method of claim 8, wherein the solid dosage form comprises a capsule. 10. The method of claim 1, wherein the migalastat is administered for at least 28 days. 11. The method of claim 1, wherein the migalastat is administered for at least 12 months. 12. The method of claim 1, wherein administration of the therapeutically effective dose of the migalastat provides an annualized rate of change in eGFRCKD-EPI of greater than −1.0 mL/min/1.73 m2. 13. The method of claim 1, wherein the therapeutically effective dose is about 123 mg FBE. 14. The method of claim 1, wherein the therapeutically effective dose is about 123 mg of migalastat free base. 15. The method of claim 1, wherein the therapeutically effective dose is about 150 mg of migalastat hydrochloride. 16. The method of claim 1, wherein the HEK assay amenable mutation in α-galactosidase A is selected from the group consisting of: L3V, L3P, R4M/Y207S, A13T, A13P, A15T, A15G, F18C, A20D, W24R, W24G, W24C, D33G, N34D, N34T, G35E, G35V, L36S, L36W, A37T, M42K, M42I, H46P, E48Q, N53D, N53L, L54F, Q57L, P60T, P60S, F69L, M721, G80D, D83N, G85S, G85N, E87D, L89F, M961, S102L, G104V, L106F, A108T, D109G, R112G, Y123C, H125L, S126G, G128E, I133M, D136E, N139S, K140T, G144D, F145S, P146S, P146R, Y152H, D165H, D165G, L166G, L167V, C174R, C174G, D175E, L180W, L180F, Y184N, K185E, p.M187_S188dup, M1871, G195V, R196G, I198T, V199A, V199G, Y200C, E203V, E203D, Y207H, M208R, P210S, P210L, K213M, P214S, P214L, N215S/D313Y, I219L, I219T, R220Q, R220P, Q221P, N224T, H225D, N228S, F229L, I232T, I242V, I242F, I242T, W245G, N249K, Q250R, Q250H, I253S, A257G, G258R, G260E, G261S, G261C, M267T, 1270M, G271S/D313Y, G271D, W277G, W277C, T282I, M284V, I289S, M290L, M290I, A291T, L294S, M296L, M296T, D299E, R301L, I303F, S304N, S304T, A307T, A309V, L311V, Q312H, D313Y, D313Y/G411D, V316I, V316G, 1319F, I319T, Q321H, D322N, D322E, P323R, G325R, K326N, Q330R, G334E, F337S, P343L, W349S, A352G, A352V, R356G, R356Q, R356P, E358Q, E358D, G360C, G361E, G361A, P362T, A368T, G375E, T385A, V390M, K391T, G395E, G395A, T412N, E418G and M421V. 17. The method of claim 1, wherein administration of the provides an about 1.8-fold increase in plasma migalastat AUC0-∞ in the patient as compared a plasma migalastat AUC0-∞ in a control subject following administration of a comparable amount of migalastat to the control subject. 18. The method of claim 1, wherein administration of the migalastat thereof provides no increase in mean plasma migalastat Cmax in the patient as compared to a plasma migalastat Cmax in a control subject following administration of a comparable amount of migalastat to the control subject. 19. The method of claim 1, wherein the patient's eGFR is from 30 to 35 mL/min/1.73 m2. 20. The method of claim 1, wherein the HEK assay amenable mutation in α-galactosidase A is selected from the group consisting of: A13T, A13P, N34D, N34T, G35V, M42K, E48Q, N53L, L54F, P60T, P60S, L89F, Y123C, H125L, I133M, K140T, F145S, P146R, D165G, L167V, L180W, K185E, R196G, V199G, Y200C, E203V, E203D, Y207H, M208R, I219L, Q221P, N224T, I242T, Q250R, Q250H, A257G, G261S, G261C, G271D, W277G, W277C, M284V, I303F, A307T, D322N, K326N, G334E, F337S, W349S, A352V, E358Q, E358D, G361E, G375E, G395E, T412N, and M421V. 21. The method of claim 1, wherein the migalastat is administered as migalastat free base. 22. The method of claim 1, wherein the migalastat is administered as a pharmaceutically acceptable salt.