Claims for Patent: 11,278,599

Summary for Patent: 11,278,599

Title:	High concentration alpha-glucosidase compositions for the treatment of Pompe disease
Abstract:	The present application provides for compositions comprising high concentrations of acid a-glucosidase in combination with an active site-specific chaperone for the acid α-glucosidase, and methods for treating Pompe disease in a subject in need thereof, that includes a method of administering to the subject such compositions. The present application also provides methods for increasing the in vitro and in vivo stability of an acid α-glucosidase enzyme formulation.
Inventor(s):	Valenzano Kenneth, Crowley John, Khanna Richie, Flanagan John
Assignee:	Amicus Therapeutics, Inc.
Application Number:	US16580541
Patent Claims:	1. A method of treating Pompe disease in a subject comprising administering(i) a first composition comprising an active site-specific chaperone selected from the group consisting of 1-deoxynojirimycin or a pharmaceutically acceptable salt thereof and n-butyl-deoxynojirimycin or a pharmaceutically acceptable salt thereof, and(ii) a second composition comprising acid α-glucosidase,wherein the acid α-glucosidase is formulated at a concentration of about 5 mg/ml to about 80 mg/ml in the second composition, andwherein the active site-specific chaperone is administered in an amount of about 1 mg/kg to about 10 mg/kg and the acid α-glucosidase is administered in an amount effective for treating Pompe disease in the subject.2. The method of claim 1 , wherein the acid α-glucosidase is administered in an amount of about 5 mg/kg to about 50 mg/kg.3. The method of claim 2 , wherein the acid α-glucosidase is administered in an amount of about 10 mg/kg to about 40 mg/kg.4. The method of claim 3 , wherein the acid α-glucosidase is administered in an amount of about 15 mg/kg to about 25 mg/kg.5. The method of claim 4 , wherein the acid α-glucosidase is administered in an amount of about 20 mg/kg.6. The method of claim 1 , wherein the second composition further comprises a buffer.7. The method of claim 6 , wherein the buffer is selected from the group consisting of citrate buffer claim 6 , acetate buffer claim 6 , bicarbonate buffer claim 6 , phosphate buffer claim 6 , and combinations thereof.8. The method of claim 1 , wherein the acid α-glucosidase is formulated at a concentration of about 15 mg/ml in the second composition.9. The method of claim 1 , wherein at least one of the first composition and the second composition further comprises an excipient.10. The method of claim 1 , wherein the active site-specific chaperone is n-butyl-deoxynojirimycin or a pharmaceutically acceptable salt thereof.11. The method of claim 1 , wherein the active site-specific chaperone is 1-deoxynojirimycin or a pharmaceutically acceptable salt thereof.12. The method of claim 1 , wherein the first composition is administered orally and the second composition is administered intravenously.13. A method of treating Pompe disease in a subject comprising administering(i) a first composition comprising n-butyl-deoxynojirimycin or a pharmaceutically acceptable salt thereof, and(ii) a second composition comprising acid α-glucosidase,wherein the acid α-glucosidase is formulated at a concentration of about 15 mg/ml in the second composition; andwherein the n-butyl-deoxynojirimycin or pharmaceutically acceptable salt thereof is administered in an amount of about 1 mg/kg to about 10 mg/kg and the acid α-glucosidase is administered in an amount of about 5 mg/kg to about 50 mg/kg.14. The method of claim 13 , wherein the acid α-glucosidase is administered in an amount of about 15 mg/kg to about 25 mg/kg.15. The method of claim 14 , wherein the acid α-glucosidase is administered in an amount of about 20 mg/kg.16. The method of claim 13 , wherein the second composition further comprises a buffer.17. The method of claim 16 , wherein the buffer is selected from the group consisting of citrate buffer claim 16 , acetate buffer claim 16 , bicarbonate buffer claim 16 , phosphate buffer claim 16 , and combinations thereof.18. The method of claim 13 , wherein the acid α-glucosidase is administered in an amount of about 10 mg/kg to about 40 mg/kg.19. The method of claim 13 , wherein at least one of the first composition and the second composition further comprises an excipient.20. The method of claim 13 , wherein the first composition is administered orally and the second composition is administered intravenously.