Claims for Patent: 12,419,937

Summary for Patent: 12,419,937

Title:	High concentration alpha-glucosidase compositions for the treatment of Pompe disease
Abstract:	The present application provides for compositions comprising high concentrations of acid α-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):	Kenneth Valenzano, John Crowley, Richie Khanna, John Flanagan
Assignee:	Amicus Therapeutics Inc , Wavefront Research Inc
Application Number:	US17/699,910
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for patent 12,419,937
Patent Claims:	1. 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 5 mg/ml to about 80 mg/mL in the second composition, and wherein the n-butyl-deoxynojirimycin 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, acetate buffer, bicarbonate buffer, phosphate buffer, and combination thereof. 8. The method of claim 1, wherein the acid α-glucosidase is formulated at a concentration of about 15 mg/kg 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 first composition is administered orally, and the second composition is administered intravenously. 11. 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; and wherein the acid α-glucosidase is administered in an amount of about 5 mg/kg to about 50 mg/kg. 12. The method of claim 11, wherein the acid α-glucosidase is administered in an amount of about 15 mg/kg to about 25 mg/kg. 13. The method of claim 12, wherein the acid α-glucosidase is administered in an amount of about 20 mg/kg. 14. The method of claim 11, wherein the second composition further comprises a buffer. 15. The method of claim 14, wherein the buffer is selected from a group consisting of citrate buffer, acetate buffer, bicarbonate buffer, phosphate buffer, and combinations thereof. 16. The method of claim 11, wherein the acid α-glucosidase is administered in an amount of about 10 mg/kg to about 40 mg/kg. 17. The method of claim 11, wherein at least one of the first composition and the second composition further comprises an excipient. 18. The method of claim 11, wherein the first composition is administered orally, and the second composition is administered intravenously.