Methods of treating Fabry patients having renal impairment
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.
1. A method of treating Fabry disease, the method comprising administering migalastat to a patient in need thereof, wherein the patient has an α-galactosidase A protein comprising a HEK assay amenable mutation selected from the group consisting of: A13P, A20D, Q57L, G80D, P146S, D175E, K213R, K213M, I242F, M267T, A309V, V316I, V316G, P323R, A352G, R356P, T385A, V390M, and G395A.
2. The method of claim 1 , wherein the mutation is selected from the group consisting of: Q57L, G80D, P146S, K213R, K213M, M267T, A309V, V316I, P323R, A352G, R356P, T385A and V390M.
3. The method of claim 1 , wherein the mutation is V316I.
4. The method of claim 1 , wherein the mutation is selected from the group consisting of: A13P, A20D, Q57L, G80D, P146S, K213R, K213M, I242F, M267T, A309V, V316I, V316G, A352G and R356P.
5. The method of claim 1 , wherein the mutation is selected from the group consisting of: A13P, A20D, G80D, P146S, I242F, M267T, V316G and R356P.
6. The method of claim 1 , wherein the mutation is selected from the group consisting of: Q57L, G80D, P146S, K213R, K213M, M267T, A309V, V316I, A352G and R356P.
7. The method of claim 1 , wherein the mutation is selected from the group consisting of: G80D, P146S, M267T and R356P.
8. The method of claim 1 , wherein the mutation is selected from the group consisting of: A13P, A20D, Q57L, G80D, P146S, D175E, K213M, I242F, M267T, A309V, V316I, V316G, P323R, A352G, R356P, T385A, V390M, and G395A.
9. The method of claim 1 , wherein the mutation is selected from the group consisting of: Q57L, G80D, P146S, K213M, M267T, A309V, V316I, P323R, A352G, R356P, T385A and V390M.
10. The method of claim 1 , wherein the mutation is selected from the group consisting of: A13P, A20D, Q57L, G80D, P146S, K213M, I242F, M267T, A309V, V316I, V316G, A352G and R356P.
11. The method of claim 1 , wherein the mutation is selected from the group consisting of: Q57L, G80D, P146S, K213M, M267T, A309V, V316I, A352G and R356P.
12. The method of claim 1 , wherein the patient has a Fabry disease-causing mutation.
13. The method of claim 1 , wherein the migalastat or salt thereof is administered to the patient every other day.
14. 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.
15. The method of claim 1 , wherein the patient is administered about 150 mg of the migalastat or salt thereof every other day.
16. The method of claim 1 , wherein the patient is administered about 150 mg of migalastat hydrochloride every other day.
17. The method of claim 1 , wherein the patient is male.
18. The method of claim 1 , wherein the patient is female.
19. The method of claim 1 , wherein the patient has renal impairment.
20. The method of claim 19 , wherein the patient has mild or moderate renal impairment.
21. The method of claim 1 , wherein the patient is an enzyme replacement therapy (ERT)-experienced patient.
22. The method of claim 1 , wherein the patient is an enzyme replacement therapy (ERT)-experienced patient with renal impairment.
23. The method of claim 1 , wherein the patient is an enzyme replacement therapy (ERT)-naïve patient.
24. 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 or salt thereof.
25. 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 or salt thereof.
26. 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 or salt thereof.
27. The method of claim 1 , wherein the migalastat or salt thereof is administered orally.
28. The method of claim 27 , wherein the migalastat or salt thereof is in a solid dosage form.
29. The method of claim 28 , wherein the solid dosage form comprises a capsule.
30. The method of claim 1 , wherein the migalastat is administered as a pharmaceutically acceptable salt.
31. The method of claim 1 , wherein the patient is orally administered a capsule comprising about 150 mg of migalastat hydrochloride every other day.
32. The method of claim 7 , wherein the patient has a Fabry disease-causing mutation.
33. The method of claim 7 , wherein the migalastat or salt thereof is administered to the patient every other day.
34. The method of claim 7 , wherein the patient is administered about 123 to about 300 mg of the migalastat or salt thereof every other day.
35. The method of claim 7 , wherein the patient is administered about 150 mg of the migalastat or salt thereof every other day.
36. The method of claim 7 , wherein the patient is administered about 150 mg of migalastat hydrochloride every other day.
37. The method of claim 7 , wherein the patient is male.
38. The method of claim 7 , wherein the patient is female.
39. The method of claim 7 , wherein the patient has renal impairment.
40. The method of claim 39 , wherein the patient has mild or moderate renal impairment.
41. The method of claim 7 , wherein the patient is an enzyme replacement therapy (ERT)-experienced patient.
42. The method of claim 7 , wherein the patient is an enzyme replacement therapy (ERT)-experienced patient with renal impairment.
43. The method of claim 7 , wherein the patient is an enzyme replacement therapy (ERT)-naïve patient.
44. The method of claim 7 , wherein the patient has a proteinuria level of less than 100 mg/24 hr prior to initiating the administration of the migalastat or salt thereof.
45. The method of claim 27 , wherein the patient has a proteinuria level of 100 to 1,000 mg/24 hr prior to initiating the administration of the migalastat or salt thereof.
46. The method of claim 7 , wherein the patient has a proteinuria level of greater than 1,000 mg/24 hr prior to initiating the administration of the migalastat or salt thereof.
47. The method of claim 7 , wherein the migalastat or salt thereof is administered orally.
48. The method of claim 47 , wherein the migalastat or salt thereof is in a solid dosage form.
49. The method of claim 48 , wherein the solid dosage form comprises a capsule.
50. The method of claim 7 , wherein the migalastat is administered as a pharmaceutically acceptable salt.
51. The method of claim 7 , wherein the patient is orally administered a capsule comprising about 150 mg of migalastat hydrochloride every other day.