IP Library › Granted Patent US 11,786,516
Granted Patent B2
US 11,786,516 · App. 18/069,720 · Granted Oct 17, 2023

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 11,786,516
App. No.
18/069,720
Granted
Oct 17, 2023
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 (19)

1. A composition comprising migalastat bound to 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 composition 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 composition of claim 1 , wherein the mutation is selected from the group consisting of: Q157H, L166S and K240N.

4. The composition 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 composition 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 composition 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 composition of claim 1 , wherein the migalastat stabilizes the α-galactosidase A protein.

8. The composition of claim 1 , wherein the composition further comprises globotriaosylceramide (GL-3).

9. The composition of claim 1 , wherein the composition comprises white blood cells containing the migalastat bound to the α-galactosidase A protein.

10. The composition of claim 1 , wherein the composition comprises plasma containing the migalastat bound to the α-galactosidase A protein.

11. The composition of claim 10 , wherein the plasma contains migalastat at a concentration in the range of about 1 ng/mL to about 2191 ng/mL.

12. The composition of claim 10 , wherein the plasma further comprises globotriaosylsphingosine (lyso-Gb3).

13. The composition of claim 12 , wherein the plasma contains lyso-Gb3 at a concentration in the range of about 9.3 to about 13.1 nM/L.

14. The composition of claim 1 , wherein the composition comprises an organ containing migalastat bound to the α-galactosidase A protein.

15. The composition of claim 14 , wherein the organ comprises a heart.

16. The composition of claim 14 , wherein the organ comprises a kidney.

17. The composition of claim 14 , wherein the organ comprises skin.

18. The composition of claim 1 , wherein the composition comprises tissue containing migalastat bound to the α-galactosidase A protein.

19. The composition of claim 1 , wherein the composition comprises cells containing migalastat bound to the α-galactosidase A protein.

Assignments (3)
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 →