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 increasing α-galactosidase A activity in a human patient, the method comprising administering migalastat to a human patient having an α-galactosidase A protein comprising a HEK assay amenable mutation 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.
2. The method of claim 1 , wherein the patient has a disease-causing mutation.
3. The method of claim 1 , wherein the migalastat or salt thereof is administered to the patient every other day.
4. The method of claim 1 , wherein the patient is administered about 123 to about 300 mg of the migalastat or salt thereof.
5. The method of claim 1 , wherein the patient is administered about 150 mg of the migalastat or salt thereof every other day.
6. The method of claim 1 , wherein the patient is administered about 150 mg of migalastat hydrochloride every other day.
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 patient is an enzyme replacement therapy (ERT)-experienced patient.
12. The method of claim 1 , wherein the patient is an enzyme replacement therapy (ERT)-experienced patient with renal impairment.
13. The method of claim 1 , wherein the patient is an enzyme replacement therapy (ERT)-naïve patient.
14. 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.
15. 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.
16. 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.
17. The method of claim 1 , wherein the migalastat or salt thereof is administered orally.
18. The method of claim 17 , wherein the migalastat or salt thereof is in a solid dosage form.
19. The method of claim 18 , wherein the solid dosage form comprises a capsule.
20. The method of claim 1 , wherein the migalastat is administered as a pharmaceutically acceptable salt.