Claims for Patent: 10,925,866

Summary for Patent: 10,925,866

Title:	Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
Abstract:	The present invention provides dosing regimens for administering pharmacological chaperones to a subject in need thereof. The dosing regimens can be used to treat disorders caused by improper protein misfolding, such as lysosomal storage disorders.
Inventor(s):	Castelli; Jeff (New Hope, PA), Lockhart; David J. (Emerald Hills, CA)
Assignee:	Amicus Therapeutics, Inc. (Cranbury, NJ)
Application Number:	16/011,063
Patent Claims:	1. A method for treatment of Fabry disease in a human patient in need thereof, the method comprising administering to the patient a therapeutically effective dose of 1-deoxygalactonojirimycin or a salt thereof, wherein the therapeutically effective dose provides one or more of: a. a mean maximum drug plasma concentration (C.sub.max) of about 9 .mu.M; b. a mean time to maximum drug plasma concentration (T.sub.max) of about 3 hours; or c. a mean drug plasma half-life (t.sub.1/2) of about 3 hours, wherein the 1-deoxygalactonojirimycin or salt thereof is administered every other day. 2. The method of claim 1, wherein the 1-deoxygalactonojirimycin or salt thereof is administered as an oral dosage form. 3. The method of claim 2, wherein the oral dosage form comprises a tablet, a capsule or a solution. 4. The method of claim 1, wherein the 1-deoxygalactonojirimycin or salt thereof enhances .alpha.-galactosidase A activity. 5. The method of claim 1, wherein the patient is male. 6. The method of claim 1, wherein the patient is female. 7. The method of claim 1, wherein the therapeutically effective dose provides a C.sub.max of about 9 .mu.M. 8. The method of claim 1, wherein the therapeutically effective dose provides a T.sub.max of about 3 hours. 9. The method of claim 1, wherein the therapeutically effective dose provides a t.sub.1/2 of about 3 hours. 10. A method for treatment of Fabry disease in a human patient in need thereof, the method comprising administering to the patient a therapeutically effective dose of 1-deoxygalactonojirimycin or a salt thereof, wherein the therapeutically effective dose provides a similar mean drug exposure (AUC) as orally administering 150 mg of migalastat hydrochloride every other day. 11. The method of claim 10, wherein the 1-deoxygalactonojirimycin or salt thereof is administered as an oral dosage form. 12. The method of claim 11, wherein the oral dosage form comprises a tablet, a capsule or a solution. 13. The method of claim 10, wherein the 1-deoxygalactonojirimycin or salt thereof is administered is administered every other day. 14. The method of claim 10, wherein the 1-deoxygalactonojirimycin or salt thereof enhances .alpha.-galactosidase A activity. 15. The method of claim 10, wherein the patient is male. 16. The method of claim 10, wherein the patient is female. 17. The method of claim 10, wherein the therapeutically effective dose provides a mean maximum drug plasma concentration (C.sub.max) of about 9 .mu.M. 18. The method of claim 10, wherein the therapeutically effective dose provides a mean time to maximum drug plasma concentration (T.sub.max) of about 3 hours. 19. The method of claim 10, wherein the therapeutically effective dose provides a mean drug plasma half-life (t.sub.1/2) of about 3 hours.