# US 11,304,940 B2 · Methods of treating Fabry patients having renal impairment

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- Number: US 11,304,940 B2
- Document ID: US11304940B2
- Status: Granted
- Kind: B2
- Application number: 17/393,971
- Filed: 2021-08-04
- Published: 2021-11-25
- Granted: 2022-04-19
- Earliest claimed: 2017-05-30
- Assignees: Amicus Therapeutics, Inc.
- Applicants: Amicus Therapeutics, Inc.
- Inventors: Jeff Castelli; Elfrida Benjamin
- Web page: https://patentgrep.com/patent/US11304940B2

## CPC

- A61K 31/445: Non condensed piperidines, e.g. piperocaine
- A61P 13/12: of the kidneys
- A61K 9/48: Preparations in capsules, e.g. of gelatin, of chocolate {(A61K9/0004 takes precedence; bite capsules A61K9/0056)}
- A61K: PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms A61J3/00; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles A61L; soap compositions C11D)
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS

## 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

### Claim 1

A molecule comprising migalastat bound to an α-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.

### Claim 2 (depends on 1)

The molecule of claim 1, wherein the mutation is selected from the group consisting of: A13T, A13P, N34D, N34T, and G35V.

### Claim 3 (depends on 1)

The molecule of claim 1, wherein the mutation is selected from the group consisting of: M42K, E48Q, N53L, L54F, P60T, and P60S.

### Claim 4 (depends on 1)

The molecule of claim 1, wherein the mutation is selected from the group consisting of: L89F, Y123C, H125L, and I133M.

### Claim 5 (depends on 1)

The molecule of claim 1, wherein the mutation is selected from the group consisting of: K140T, F145S, P146R, D165G, L167V, L180W, and K185E.

### Claim 6 (depends on 1)

The molecule of claim 1, wherein the mutation is selected from the group consisting of: R196G, V199G, Y200C, E203V, E203D, Y207H, and M208R.

### Claim 7 (depends on 1)

The molecule of claim 1, wherein the mutation is selected from the group consisting of: I219L, Q221P, N224T, I242T, Q250R, and Q250H.

### Claim 8 (depends on 1)

The molecule of claim 1, wherein the mutation is selected from the group consisting of: A257G, G261S, G261C, G271D, W277G, W277C, and M284V.

### Claim 9 (depends on 1)

The molecule of claim 1, wherein the mutation is selected from the group consisting of: I303F and A307T.

### Claim 10 (depends on 1)

The molecule of claim 1, wherein the mutation is selected from the group consisting of: D322N, K326N, G334E, F337S, W349S, and A352V.

### Claim 11 (depends on 1)

The molecule of claim 1, wherein the mutation is selected from the group consisting of: E358Q, E358D, G361E, G375E, G395E, T412N, and M421V.

### Claim 12

An α-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 α-galactosidase A protein having the same mutation.

### Claim 13 (depends on 12)

The α-galactosidase A protein of claim 12, wherein the protein is bound to migalastat.

### Claim 14 (depends on 12)

The α-galactosidase A protein of claim 12, wherein the mutation is selected from the group consisting of: A13T, A13P, N34D, N34T, and G35V.

### Claim 15 (depends on 12)

The α-galactosidase A protein of claim 12, wherein the mutation is selected from the group consisting of: M42K, E48Q, N53L, L54F, P60T, and P60S.

### Claim 16 (depends on 12)

The α-galactosidase A protein of claim 12, wherein the mutation is selected from the group consisting of: L89F, Y123C, H125L, and I133M.

### Claim 17 (depends on 12)

The α-galactosidase A protein of claim 12, wherein the mutation is selected from the group consisting of: K140T, F145S, P146R, D165G, L167V, L180W, and K185E.

### Claim 18 (depends on 12)

The α-galactosidase A protein of claim 12, wherein the mutation is selected from the group consisting of: R196G, V199G, Y200C, E203V, E203D, Y207H, and M208R.

### Claim 19 (depends on 12)

The α-galactosidase A protein of claim 12, wherein the mutation is selected from the group consisting of: I219L, Q221P, N224T, I242T, Q250R, and Q250H.

### Claim 20 (depends on 12)

The α-galactosidase A protein of claim 12, wherein the mutation is selected from the group consisting of: A257G, G261S, G261C, G271D, W277G, W277C, and M284V.

### Claim 21 (depends on 12)

The α-galactosidase A protein of claim 12, wherein the mutation is selected from the group consisting of: I303F and A307T.

### Claim 22 (depends on 12)

The α-galactosidase A protein of claim 12, wherein the mutation is selected from the group consisting of: D322N, K326N, G334E, F337S, W349S, and A352V.

### Claim 23 (depends on 12)

The α-galactosidase A protein of claim 12, wherein the mutation is selected from the group consisting of: E358Q, E358D, G361E, G375E, G395E, T412N, and M421V.