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 a Fabry patient, the method comprising administering migalastat to a Fabry patient having an α-galactosidase A protein comprising a HEK assay amenable mutation selected from the group consisting of D33G, L36W, Q57L, M96I, R112G, C174R, M187I, I253S, G258R, G271S/D313Y, D313Y, D322E, G325R, and R356Q.
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.