IP Library Granted Patent US 12,042,488
Granted Patent B2
US 12,042,488 · App. 18/326,274 · 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 12,042,488
App. No.
18/326,274
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 (10)

1. An α-galactosidase A protein having a HEK amenable mutation selected from the group consisting of: G334E, N34D and p.V254del, wherein the protein is bound to migalastat.

2. The α-galactosidase A protein of claim 1 , wherein the mutation is G334E.

3. The α-galactosidase A protein of claim 1 , wherein the mutation is N34D.

4. The α-galactosidase A protein of claim 1 , wherein the mutation is p.V254del.

5. The α-galactosidase A protein of claim 1 , wherein the protein bound to migalastat has increased stability as compared to a naturally-occurring α-galactosidase A protein having the same mutation.

6. The α-galactosidase A protein of claim 5 , wherein the mutation is G334E.

7. The α-galactosidase A protein of claim 5 , wherein the mutation is N34D.

8. The α-galactosidase A protein of claim 5 , wherein the mutation is p.V254del.

9. An α-galactosidase A protein having a HEK amenable mutation N215I, wherein the protein is bound to migalastat.

10. The α-galactosidase A protein of claim 9 , wherein the protein bound to migalastat has increased stability as compared to a naturally-occurring α-galactosidase A protein having the same mutation.

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 →