# US 9,750,733 B2 · Compounds for preventing and/or treating lysosomal storage disorders and/or degenerative disorders of the central nervous system

> Agents: this data is available as JSON from the patentgrep API and MCP server — see https://patentgrep.com/llms.txt

- Number: US 9,750,733 B2
- Document ID: US9750733B2
- Status: Granted
- Kind: B2
- Application number: 15/289,283
- Filed: 2016-10-10
- Published: 2017-01-26
- Granted: 2017-09-05
- Earliest claimed: 2012-03-27
- Assignees: Amicus Therapeutics, Inc.
- Applicants: Amicus Therapeutics, Inc.
- Inventors: Robert Boyd; Philip J. Rybczynski; Kamlesh Sheth
- Web page: https://patentgrep.com/patent/US9750733B2

## CPC

- H10B 12/0335: {Making a connection between the transistor and the capacitor, e.g. plug}
- A61K 31/445: Non condensed piperidines, e.g. piperocaine
- A61K 45/06: Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
- A61P 25/00: Drugs for disorders of the nervous system
- C07D 211/44: attached in position 4
- H10B 12/033: {the capacitor extending over the transistor}
- H10B 12/05: {Making the transistor}
- H10B 12/09: {with simultaneous manufacture of the peripheral circuit region and memory cells}
- H10B 12/315: {with the capacitor higher than a bit line}
- H10D 1/716: {having vertical extensions}
- H10P 50/283: {by chemical means}
- H10P 50/71: {using masks for conductive or resistive materials}
- H10B: ELECTRONIC MEMORY DEVICES
- 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
- C07D: HETEROCYCLIC COMPOUNDS (macromolecular compounds C08)
- H10D: INORGANIC ELECTRIC SEMICONDUCTOR DEVICES
- H10P: GENERIC PROCESSES OR APPARATUS FOR THE MANUFACTURE OR TREATMENT OF DEVICES COVERED BY CLASS H10

## Abstract

Described are novel salts of the compound (3R,4R,5S)-5-(difluoromethyl) piperidine-3,4-diol, as well as methods of using the same for preventing and/or treating lysosomal storage disorders and/or degenerative disorders of the central nervous system. In particular, the present invention provides methods for preventing and/or treating Gaucher's disease and/or Parkinson's disease.

## Claims

### Claim 1

A method for treating Parkinson's disease in a patient at risk for developing or diagnosed with the same, which comprises administering to the patient an effective amount of a crystalline form of (3R,4R,5S)-5-(difluoromethyl)piperidine-3,4-diol•fumarate (1:1), wherein the crystalline form has an X-ray diffraction pattern comprising characteristic peaks at:
2-Theta (Degrees)
11.70 ± 0.2
15.70 ± 0.2
20.78 ± 0.2
21.46 ± 0.2
27.74 ± 0.2.

### Claim 2 (depends on 1)

The method of claim 1, wherein the crystalline form has an X-ray diffraction pattern comprising characteristic peaks at:
2-Theta (Degrees)
11.70 ± 0.2
14.66 ± 0.2
15.70 ± 0.2
19.34 ± 0.2
20.78 ± 0.2
21.46 ± 0.2
22.22 ± 0.2
26.26 ± 0.2
26.90 ± 0.2
27.74 ± 0.2.

### Claim 3 (depends on 1)

The method of claim 1, wherein the crystalline form has an X-ray diffraction pattern of FIG. 1.

### Claim 4 (depends on 1)

The method of claim 1, wherein the crystalline form is administered as part of a pharmaceutical composition comprising a pharmaceutically acceptable carrier.

### Claim 5

A method for treating Gaucher's disease in a patient at risk for developing or diagnosed with the same, which comprises administering to the patient an effective amount of a crystalline form of (3R,4R,5S)-5-(difluoromethyl)piperidine-3,4-diol.fumarate (1:1), wherein the crystalline form has an X-ray diffraction pattern comprising characteristic peaks at:
2-Theta (Degrees)
11.70 ± 0.2
15.70 ± 0.2
20.78 ± 0.2
21.46 ± 0.2
27.74 ± 0.2.

### Claim 6 (depends on 5)

The method of claim 5, wherein the crystalline form has an X-ray diffraction pattern comprising characteristic peaks at:
2-Theta (Degrees)
11.70 ± 0.2
14.66 ± 0.2
15.70 ± 0.2
19.34 ± 0.2
20.78 ± 0.2
21.46 ± 0.2
22.22 ± 0.2
26.26 ± 0.2
26.90 ± 0.2
27.74 ± 0.2.

### Claim 7 (depends on 5)

The method of claim 5, wherein the crystalline form has an X-ray diffraction pattern of FIG. 1.

### Claim 8 (depends on 5)

The method of claim 5, wherein the crystalline form is administered as part of a pharmaceutical composition comprising a pharmaceutically acceptable carrier.