IP Library › Granted Patent US 12,465,586
Granted Patent B2
US 12,465,586 · App. 17/865,195 · Granted Nov 11, 2025

Inhibitors of glucosylceramide synthase

Inventors: Hanlan Liu (Lexington, MA); Chris Willis (Southborough, MA); Renu Bhardwaj (Ashland, MA); Jianmei Kochling (Wellesley, MA); Judith Peterschmitt (Watertown, MA); Craig Siegel (Woburn, MA)
Assignee: Genzyme Corporation
A61K31/4025A61K31/357A61K45/06A61P3/00A61P3/06
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Quick Facts
Patent No.
US 12,465,586
App. No.
17/865,195
Granted
Nov 11, 2025
Kind
B2
Abstract

The hemitartrate salt of a compound represented by the following structural formula: (Formula I Hemitartrate), which may be used in pharmaceutical applications, are disclosed. Particular single crystalline forms of the Formula (I) Hemitartrate are characterized by a variety of properties and physical measurements. As well, methods of producing crystalline Formula (I) Hemitartrate, and using it to inhibit glucosylceramide synthase or lowering glycosphingolipid concentrations in subjects to treat a number of diseases, are also discussed. Pharmaceutical compositions are also described.

Claims (10)

1 . A method of treating a human patient with type 1 Gaucher disease who has been assessed as being a poor CYP2D6 P450 metabolizer as determined by CYP2D6 genotyping, comprising administering to said patient the subject an effective amount of a hemitartrate salt of the compound of formula (I)

wherein the effective amount is 100 milligrams, wherein the hemitartrate salt of the compound of formula (I) is characterized by at least three X-ray powder diffraction peaks at 2θ angles selected from the group consisting of 5.1°, 6.6°, 10.7°, 11.0°, 15.9°, and 21.7°, wherein the specified 2θ angle means the specified value±0.2°, and wherein the X-ray powder diffraction peaks are obtained by using Cu Kα radiation.

2 . The method of claim 1 , wherein the effective amount of the hemitartrate salt of the compound of formula (I) is administered in an oral dosage form once per day.

3 . The method of claim 2 , wherein the oral dosage form is a capsule.

4 . A method of treating a human patient with type 1 Gaucher disease who has been assessed as being an intermediate CYP2D6 P450 metabolizer as determined by CYP2D6 genotyping comprising administering to said patient an effective amount of a hemitartrate salt of the compound of formula (I)

wherein the effective amount is 100 milligrams administered in an oral dosage form twice per day, wherein the hemitartrate salt of the compound of formula (I) is characterized by at least three X-ray powder diffraction peaks at 2θ angles selected from the group consisting of 5.1°, 6.6°, 10.7°, 11.0°, 15.9°, and 21.7°, wherein the specified 2θ angle means the specified value±0.2°, and wherein the X-ray powder diffraction peaks are obtained by using Cu Kα radiation.

5 . The method of claim 4 , wherein the oral dosage form is a capsule.

6 . A method of treating a human patient with type 1 Gaucher disease who has been assessed as being an extensive CYP2D6 P450 metabolizer as determined by CYP2D6 genotyping comprising administering to said patient an effective amount of a hemitartrate salt of the compound of formula (I)

wherein the effective amount is 100 milligrams administered in an oral dosage form twice per day, wherein the hemitartrate salt of the compound of formula (I) is characterized by at least three X-ray powder diffraction peaks at 2θ angles selected from the group consisting of 5.1°, 6.6°, 10.7°, 11.0°, 15.9°, and 21.7°, wherein the specified 2θ angle means the specified value±0.2°, and wherein the X-ray powder diffraction peaks are obtained by using Cu Kα radiation.

7 . The method of claim 6 , wherein the oral dosage form is a capsule.

Assignments (1)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Mar 7, 2023
From: LIU, HANLAN; WILLIS, CHRISTOPHER; BHARDWAJ, RENU; KOCHLING, JIANMEI; PETERSCHMITT, JUDITH; SIEGEL, CRAIG
To: GENZYME CORPORATION
Reel/Frame 062905/0567 →
Continuity (3)
Division 13511768
Provisional Application 61264748 · Nov 27, 2009
Related Publication 20230172903A1 · Jun 8, 2023
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