IP Library › Granted Patent US 11,376,244
Granted Patent B2
US 11,376,244 · App. 17/670,095 · Granted Jul 5, 2022

Methods of treating Fabry patients having renal impairment

Inventors: Jeff Castelli (New Hope, PA); Elfrida Benjamin (Millstone Township, NJ)
Assignee: Amicus Therapeutics, Inc.
A61K31/445A61P13/12A61K9/48
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Quick Facts
Patent No.
US 11,376,244
App. No.
17/670,095
Granted
Jul 5, 2022
Kind
B2
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.

Claims (31)

1. A molecule comprising migalastat bound to 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 molecule of claim 1 , wherein the mutation is D33G.

3. The molecule of claim 1 , wherein the mutation is L36W.

4. The molecule of claim 1 , wherein the mutation is Q57L.

5. The molecule of claim 1 , wherein the mutation is M96I.

6. The molecule of claim 1 , wherein the mutation is R112G.

7. The molecule of claim 1 , wherein the mutation is C174R.

8. The molecule of claim 1 , wherein the mutation is M187I.

9. The molecule of claim 1 , wherein the mutation is I253S.

10. The molecule of claim 1 , wherein the mutation is G258R.

11. The molecule of claim 1 , wherein the mutation is G271S/D313Y or D313Y.

12. The molecule of claim 1 , wherein the mutation is D322E.

13. The molecule of claim 1 , wherein the mutation is G325R.

14. The molecule of claim 1 , wherein the mutation is R356Q.

15. The molecule of claim 1 , wherein the mutation is a disease-causing mutation.

16. An α-galactosidase A protein having a HEK amenable mutation selected from the group consisting of D33G, L36W, Q57L, M96I, R112G, C174R, M187I, I253S, G258R, G271S/D313Y, D313Y, D322E, G325R, and R356Q,

wherein the protein has increased stability as compared to a naturally-occurring α-galactosidase A protein having the same mutation.

17. The α-galactosidase A protein of claim 16 , wherein the protein is bound to migalastat.

18. The α-galactosidase A protein of claim 16 , wherein the mutation is D33G.

19. The α-galactosidase A protein of claim 16 , wherein the mutation is L36W.

20. The α-galactosidase A protein of claim 16 , wherein the mutation is Q57L.

21. The α-galactosidase A protein of claim 16 , wherein the mutation is M96I.

22. The α-galactosidase A protein of claim 16 , wherein the mutation is R112G.

23. The α-galactosidase A protein of claim 16 , wherein the mutation is C174R.

24. The α-galactosidase A protein of claim 16 , wherein the mutation is M187I.

25. The α-galactosidase A protein of claim 16 , wherein the mutation is I253S.

26. The α-galactosidase A protein of claim 16 , wherein the mutation is G258R.

27. The α-galactosidase A protein of claim 16 , wherein the mutation is G271S/D313Y or D313Y.

28. The α-galactosidase A protein of claim 16 , wherein the mutation is D322E.

29. The α-galactosidase A protein of claim 16 , wherein the mutation is G325R.

30. The α-galactosidase A protein of claim 16 , wherein the mutation is R356Q.

Assignments (4)
SECURITY INTEREST Recorded Apr 27, 2026
From: BIOMARIN PHARMACEUTICAL INC.; AMICUS THERAPEUTICS, INC.
To: CITIBANK, N.A., AS COLLATERAL AGENT
Reel/Frame 075493/0968 →
RELEASE OF SECURITY INTEREST Recorded Apr 27, 2026
From: WILMINGTON TRUST, NATIONAL ASSOCIATION
To: AMICUS THERAPEUTICS, INC.
Reel/Frame 075494/0030 →
SECURITY INTEREST Recorded Oct 6, 2023
From: AMICUS THERAPEUTICS, INC.
To: WILMINGTON TRUST, NATIONAL ASSOCIATION
Reel/Frame 065177/0196 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Aug 5, 2022
From: CASTELLI, JEFF; BENJAMIN, ELFRIDA
To: AMICUS THERAPEUTICS, INC.
Reel/Frame 061099/0803 →
Cited By (1)
US 12,594,268