IP Library Granted Patent US 9,464,050
Granted Patent B2
US 9,464,050 · App. 14/387,646 · Granted Oct 11, 2016

Compounds for preventing and/or treating lysosomal storage disorders and/or degenerative disorders of the central nervous system

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Quick Facts
Patent No.
US 9,464,050
App. No.
14/387,646
Granted
Oct 11, 2016
Kind
B2
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 (25)

1. 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.

2. The crystalline form of claim 1 having 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.

3. A crystalline form of (3R,4R,5S)-5-(difluoromethyl)piperidine-3,4-diol•fumarate (1:1) having an X-ray diffraction pattern of FIG. 1 .

4. A pharmaceutical composition comprising the crystalline form of claim 1 and at least one pharmaceutically acceptable carrier.

5. A crystalline form of (3R,4R,5S)-5-(difluoromethyl)piperidine-3,4-diol•fumarate (1:1) made by a process comprising:

preparing a solution comprising (3R,4R,5S)-5-(difluoromethyl)piperidine-3,4-diol, fumaric acid and a solvent, wherein the solvent is ethanol, ethyl acetate, ethyl formate, acetone, 2-butanone, dichloromethane, tetrahydrofuran, water or acetonitrile; and

crystallizing the (3R,4R,5S)-5-(difluoromethyl)piperidine-3,4-diol•fumarate (1:1) from the solution.

6. The crystalline form of claim 5 , wherein the solvent includes one or more of ethanol and water.

Assignments (4)
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 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Nov 24, 2014
From: BOYD, ROBERT; RYBCZYNSKI, PHILIP J.; SHETH, KAMLESH
To: AMICUS THERAPEUTICS, INC.
Reel/Frame 034250/0199 →