IP Library Granted Patent US 12,042,490
Granted Patent B2
US 12,042,490 · App. 18/326,281 · Granted Jul 23, 2024

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 12,042,490
App. No.
18/326,281
Granted
Jul 23, 2024
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 (14)

1. A method of treating Fabry disease, the method comprising administering 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: I242F, G334E, N34D and p.V254del.

2. The method of claim 1 , wherein the mutation is selected from the group consisting of G334E, N34D and p.V254del.

3. The method of claim 1 , wherein the mutation is I242F.

4. The method of claim 1 , wherein the mutation is G334E.

5. The method of claim 1 , wherein the mutation is N34D.

6. The method of claim 1 , wherein the mutation is p.V254del.

7. The method of claim 1 , wherein the patient is administered about 150 mg of migalastat hydrochloride every other day.

8. The method of claim 7 , wherein the mutation is selected from the group consisting of G334E, N34D and p.V254del.

9. The method of claim 7 , wherein the mutation is I242F.

10. The method of claim 7 , wherein the mutation is G334E.

11. The method of claim 7 , wherein the mutation is N34D.

12. The method of claim 7 , wherein the mutation is p.V254del.

13. A method of treating Fabry disease, the method comprising administering migalastat to a patient in need thereof, wherein the patient has an α-galactosidase A protein comprising a HEK assay amenable mutation N215I.

14. The method of claim 13 , wherein the patient is administered about 150 mg of migalastat hydrochloride every other day.

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 →