You’re using a public version of DrugPatentWatch with 5 free searches available | Register to unlock more free searches. CREATE FREE ACCOUNT

Last Updated: May 19, 2024

Claims for Patent: 11,304,940

✉ Email this page to a colleague

« Back to Dashboard

Summary for Patent: 11,304,940

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 .alpha.-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):	Castelli; Jeff (New Hope, PA), Benjamin; Elfrida (Millstone Township, NJ)
Assignee:	Amicus Therapeutics, Inc. (Philadelphia, PA)
Application Number:	17/393,971
Patent Claims:	1. A molecule comprising migalastat bound to an .alpha.-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 molecule of claim 1, wherein the mutation is selected from the group consisting of: A13T, A13P, N34D, N34T, and G35V. 3. The molecule of claim 1, wherein the mutation is selected from the group consisting of: M42K, E48Q, N53L, L54F, P60T, and P60S. 4. The molecule of claim 1, wherein the mutation is selected from the group consisting of: L89F, Y123C, H125L, and I133M. 5. The molecule of claim 1, wherein the mutation is selected from the group consisting of: K140T, F145S, P146R, D165G, L167V, L180W, and K185E. 6. The molecule of claim 1, wherein the mutation is selected from the group consisting of: R196G, V199G, Y200C, E203V, E203D, Y207H, and M208R. 7. The molecule of claim 1, wherein the mutation is selected from the group consisting of: I219L, Q221P, N224T, I242T, Q250R, and Q250H. 8. The molecule of claim 1, wherein the mutation is selected from the group consisting of: A257G, G261S, G261C, G271D, W277G, W277C, and M284V. 9. The molecule of claim 1, wherein the mutation is selected from the group consisting of: I303F and A307T. 10. The molecule of claim 1, wherein the mutation is selected from the group consisting of: D322N, K326N, G334E, F337S, W349S, and A352V. 11. The molecule of claim 1, wherein the mutation is selected from the group consisting of: E358Q, E358D, G361E, G375E, G395E, T412N, and M421V. 12. An .alpha.-galactosidase A protein having a HEK 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, wherein the protein has increased stability as compared to a naturally-occurring .alpha.-galactosidase A protein having the same mutation. 13. The .alpha.-galactosidase A protein of claim 12, wherein the protein is bound to migalastat. 14. The .alpha.-galactosidase A protein of claim 12, wherein the mutation is selected from the group consisting of: A13T, A13P, N34D, N34T, and G35V. 15. The .alpha.-galactosidase A protein of claim 12, wherein the mutation is selected from the group consisting of: M42K, E48Q, N53L, L54F, P60T, and P60S. 16. The .alpha.-galactosidase A protein of claim 12, wherein the mutation is selected from the group consisting of: L89F, Y123C, H125L, and I133M. 17. The .alpha.-galactosidase A protein of claim 12, wherein the mutation is selected from the group consisting of: K140T, F145S, P146R, D165G, L167V, L180W, and K185E. 18. The .alpha.-galactosidase A protein of claim 12, wherein the mutation is selected from the group consisting of: R196G, V199G, Y200C, E203V, E203D, Y207H, and M208R. 19. The .alpha.-galactosidase A protein of claim 12, wherein the mutation is selected from the group consisting of: I219L, Q221P, N224T, I242T, Q250R, and Q250H. 20. The .alpha.-galactosidase A protein of claim 12, wherein the mutation is selected from the group consisting of: A257G, G261S, G261C, G271D, W277G, W277C, and M284V. 21. The .alpha.-galactosidase A protein of claim 12, wherein the mutation is selected from the group consisting of: I303F and A307T. 22. The .alpha.-galactosidase A protein of claim 12, wherein the mutation is selected from the group consisting of: D322N, K326N, G334E, F337S, W349S, and A352V. 23. The .alpha.-galactosidase A protein of claim 12, wherein the mutation is selected from the group consisting of: E358Q, E358D, G361E, G375E, G395E, T412N, and M421V.

Make Better Decisions: Try a trial or see plans & pricing

Drugs may be covered by multiple patents or regulatory protections. All trademarks and applicant names are the property of their respective owners or licensors. Although great care is taken in the proper and correct provision of this service, thinkBiotech LLC does not accept any responsibility for possible consequences of errors or omissions in the provided data. The data presented herein is for information purposes only. There is no warranty that the data contained herein is error free. thinkBiotech performs no independent verification of facts as provided by public sources nor are attempts made to provide legal or investing advice. Any reliance on data provided herein is done solely at the discretion of the user. Users of this service are advised to seek professional advice and independent confirmation before considering acting on any of the provided information. thinkBiotech LLC reserves the right to amend, extend or withdraw any part or all of the offered service without notice.