Methods for treatment of Fabry disease

Patent metadata

Field	Value
Publication number	US-9480682-B2
Application number	US-201514641707-A
Country	US
Kind code	B2
Filing date	Mar 9, 2015
Priority date	May 16, 2006
Publication date	Nov 1, 2016
Grant date	Nov 1, 2016

Abstract

Provided are in vitro and in vivo methods for determining whether a patient with Fabry disease will respond to treatment with a specific pharmacological chaperone.

First claim

What is claimed is:

1. A method for treatment of Fabry disease in a patient in need thereof, the method comprising administering to the patient a formulation comprising 150 mg of 1-deoxygalactonojirimycin or a salt thereof every other day.

2. The method of claim 1 , wherein the formulation is administered for at least 2 weeks.

3. The method of claim 1 , wherein the salt of 1-deoxygalactonojirimycin comprises migalastat hydrochloride.

4. The method of claim 1 , wherein the formulation comprises an oral dosage form.

5. The method of claim 4 , wherein the oral dosage form comprises a tablet, a capsule or a solution.

6. The method of claim 1 , wherein the formulation comprises an injectable solution.

7. The method of claim 6 , wherein the injectable solution comprises a carrier comprising water, an aqueous saline solution, an aqueous dextrose solution or an aqueous glycerol solution.

8. The method of claim 7 , wherein the carrier comprises a sterile liquid.

9. The method of claim 1 , wherein the 1-deoxygalactonojirimycin or salt thereof enhances α-galactosidase A activity.

10. A method for treatment of Fabry disease in a patient in need thereof, the method comprising administering to the patient a formulation comprising 1-deoxygalactonojirimycin or a salt thereof, wherein the 1-deoxygalactonojirimycin or salt thereof is administered to the patient in an amount of 150 mg every other day for at least 2 weeks.

11. The method of claim 10 , wherein the 1-deoxygalactonojirimycin or salt thereof is administered orally.

12. The method of claim 10 , wherein the 1-deoxygalactonojirimycin or salt thereof is administered by injection.

13. The method of claim 10 , wherein the salt of 1-deoxygalactonojirimycin comprises migalastat hydrochloride.

14. The method of claim 10 , wherein the 1-deoxygalactonojirimycin or salt thereof enhances α-galactosidase A activity.

Assignees

• Amicus Therapeutics Inc

Inventors

• Lockhart David
• Castelli Jeff

Classifications

• A61P9/00

  Drugs for disorders of the cardiovascular system · CPC title

• A61P9/10

  for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis · CPC title

• A61P43/00

  Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00 · CPC title

• A61P3/00

  Drugs for disorders of the metabolism (of the blood or the extracellular fluid A61P7/00) · CPC title

• A61P13/12

  of the kidneys · CPC title