Claims for Patent: 12,280,042

Summary for Patent: 12,280,042

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 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.
Inventor(s):	Jeff Castelli, Elfrida Benjamin
Assignee:	Amicus Therapeutics Inc
Application Number:	US18/069,732
Patent Claims:	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.