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Last Updated: October 29, 2020

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Claims for Patent: 10,471,053

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Summary for Patent: 10,471,053
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. (Cranbury, NJ)
Application Number:16/284,582
Patent Claims: 1. A method for treatment of Fabry disease in a human patient in need thereof, the method comprising administering to the patient a therapeutically effective dose of migalastat or a salt thereof, wherein the patient has a HEK assay amenable mutation in .alpha.-galactosidase A 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, M72I, G80D, D83N, G85S, G85N, E87D, L89F, M96I, 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, M187I, 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, I270M, 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, I319F, 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.

2. The method of claim 1, wherein the migalastat or salt thereof is administered to the patient every other day.

3. The method of claim 1, wherein the patient is administered about 123 to about 300 mg of the migalastat or salt thereof every other day.

4. The method of claim 1, wherein the patient is administered about 150 mg of the migalastat or salt thereof every other day.

5. The method of claim 1, wherein the patient is administered about 150 mg of migalastat hydrochloride every other day.

6. The method of claim 1, wherein the migalastat or salt thereof enhances .alpha.-galactosidase A activity.

7. The method of claim 1, wherein the patient is male.

8. The method of claim 1, wherein the patient is female.

9. The method of claim 1, wherein the patient has renal impairment.

10. The method of claim 9, wherein the patient has mild or moderate renal impairment.

11. The method of claim 1, wherein the mutation is selected from the group consisting of: L3V, L3P, R4M/Y207S, A13T, A13P, A15T, A15G, F18C, A20D, W24R, W24G, W24C, D33G, N34D, N34T, G35E and G35V.

12. The method of claim 1, wherein the mutation is selected from the group consisting of: L36S, L36W, A37T, M42K, M42I, H46P, E48Q, N53D, N53L, L54F, Q57L, P60T, P60S, F69L, M72I, G80D and D83N.

13. The method of claim 1, wherein the mutation is selected from the group consisting of: G85S, G85N, E87D, L89F, M96I, S102L, G104V, L106F, A108T, D109G, R112G, Y123C, H125L, S126G, G128E, I133M, D136E and N139S.

14. The method of claim 1, wherein the mutation is selected from the group consisting of: K140T, G144D, F145S, P146S, P146R, Y152H, D165H, D165G, L166G, L167V, C174R, C174G, D175E, L180W, L180F, Y184N and K185E.

15. The method of claim 1, wherein the mutation is selected from the group consisting of: p.M187_S188dup, M187I, G195V, R196G, I198T, V199A, V199G, Y200C, E203V, E203D, Y207H, M208R, P210S, P210L, K213M, P214S, P214L and N215S/D313Y.

16. The method of claim 1, wherein the mutation is selected from the group consisting of: I219L, I219T, R220Q, R220P, Q221P, N224T, H225D, N228S, F229L, I232T, I242V, I242F, I242T, W245G, N249K, Q250R and Q250H.

17. The method of claim 1, wherein the mutation is selected from the group consisting of: I253S, A257G, G258R, G260E, G261S, G261C, M267T, I270M, G271S/D313Y, G271D, W277G, W277C, T282I, M284V, I289S, M290L and M290I.

18. The method of claim 1, wherein the mutation is selected from the group consisting of: A291T, L294S, M296L, M296T, D299E, R301L, I303F, S304N, S304T, A307T, A309V, L311V, Q312H, D313Y, D313Y/G411D, V316I and V316G.

19. The method of claim 1, wherein the mutation is selected from the group consisting of: I319F, I319T, Q321H, D322N, D322E, P323R, G325R, K326N, Q330R, G334E, F337S, P343L, W349S, A352G, A352V, R356G, R356Q and R356P.

20. The method of claim 1, wherein the mutation is selected from the group consisting of: E358Q, E358D, G360C, G361E, G361A, P362T, A368T, G375E, T385A, V390M, K391T, G395E, G395A, T412N, E418G and M421V.

21. A method for treatment of Fabry disease in a human patient in need thereof, the method comprising administering to the patient a therapeutically effective dose of migalastat or a salt thereof, wherein the patient has a HEK assay amenable mutation in .alpha.-galactosidase A disclosed in a reference table, wherein the reference table includes one or more of the following mutations: 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, M72I, G80D, D83N, G85S, G85N, E87D, L89F, M96I, 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, M187I, 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, I270M, 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, I319F, 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 or M421V.

22. The method of claim 21, wherein the reference table includes each of the following mutations: 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, M72I, G80D, D83N, G85S, G85N, E87D, L89F, M96I, 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, M187I, 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, I270M, 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, I319F, 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.

23. The method of claim 21, wherein the migalastat or salt thereof is administered to the patient every other day.

24. The method of claim 21, wherein the patient is administered about 123 to about 300 mg of the migalastat or salt thereof every other day.

25. The method of claim 21, wherein the patient is administered about 150 mg of the migalastat or salt thereof every other day.

26. The method of claim 21, wherein the patient is administered about 150 mg of migalastat hydrochloride every other day.

27. The method of claim 21, wherein the migalastat or salt thereof enhances .alpha.-galactosidase A activity.

28. The method of claim 21, wherein the patient is male.

29. The method of claim 21, wherein the patient is female.

30. The method of claim 21, wherein the patient has renal impairment.

31. The method of claim 30, wherein the patient has mild or moderate renal impairment.

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