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 a formulation comprising 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: D55G, H125Y, Q157H, L166S, N215I, K240N, G261R, L275V, N278Y, W287L, K308E and P343T.
2. The method of claim 1 , wherein the mutation is selected from the group consisting of: D55G, Q157H, L166S, N215I, K240N, G261R, L275V, N278Y, K308E and P343T.
3. The method of claim 1 , wherein the mutation is selected from the group consisting of: Q157H, L166S and K240N.
4. The method of claim 1 , wherein the mutation is selected from the group consisting of: D55G, H125Y, Q157H, L166S, N215I, K240N, G261R, L275V, N278Y, W287L and K308E.
5. The method of claim 1 , wherein the mutation is selected from the group consisting of: D55G, H125Y, Q157H, L166S, K240N, G261R, L275V, N278Y, W287L and K308E.
6. The method of claim 1 , wherein the mutation is selected from the group consisting of: D55G, Q157H, L166S, N215I, K240N, G261R, L275V, N278Y and K308E.
7. The method of claim 1 , wherein the patient has a Fabry disease-causing mutation.
8. The method of claim 1 , wherein the migalastat is administered to the patient every other day.
9. The method of claim 1 , wherein the patient is administered about 123 to about 300 mg of the migalastat every other day.
10. The method of claim 1 , wherein the patient is administered about 150 mg of the migalastat every other day.
11. The method of claim 1 , wherein the patient is administered about 150 mg of migalastat hydrochloride every other day.
12. The method of claim 1 , wherein the patient is male.
13. The method of claim 1 , wherein the patient is female.
14. The method of claim 1 , wherein the patient has renal impairment.
15. The method of claim 14 , wherein the patient has mild or moderate renal impairment.
16. The method of claim 1 , wherein the patient is an enzyme replacement therapy (ERT)-experienced patient.
17. The method of claim 1 , wherein the patient is an enzyme replacement therapy (ERT)-experienced patient with renal impairment.
18. The method of claim 1 , wherein the patient is an enzyme replacement therapy (ERT)-naïve patient.
19. 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.
20. 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.
21. 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.
22. The method of claim 1 , wherein the migalastat is administered orally.
23. The method of claim 22 , wherein the migalastat is in a solid dosage form.
24. The method of claim 23 , wherein the solid dosage form comprises a capsule.
25. The method of claim 1 , wherein the migalastat is administered as a pharmaceutically acceptable salt.
26. The method of claim 1 , wherein the patient is orally administered a capsule comprising about 150 mg of migalastat hydrochloride every other day.
27. The method of claim 1 , wherein the mutation is selected from the group consisting of Q157H, K240N, L275V, N278Y and W287L.
28. The method of claim 1 , wherein the mutation is selected from the group consisting of: D55G, G261R, L275V, N278Y and K308E.
29. The method of claim 1 , wherein the mutation is selected from the group consisting of: G261R and L275V.
30. A molecule comprising migalastat bound to an α-galactosidase A protein comprising a HEK assay amenable mutation selected from the group consisting of: Q157H, K240N, L275V, N278Y and W287L.
31. The molecule of claim 30 , wherein the protein bound to migalastat has increased stability as compared to a naturally-occurring α-galactosidase A protein having the same mutation.
32. A molecule comprising migalastat bound to an α-galactosidase A protein comprising a HEK assay amenable mutation selected from the group consisting of: D55G, G261R, L275V, N278Y and K308E.
33. The molecule of claim 32 , wherein the protein bound to migalastat has increased stability as compared to a naturally-occurring α-galactosidase A protein having the same mutation.
34. The molecule of claim 32 , wherein the mutation is selected from the group consisting of: G261R and L275V.
35. The molecule of claim 34 , wherein the protein bound to migalastat has increased stability as compared to a naturally-occurring α-galactosidase A protein having the same mutation.