Claims for Patent: 10,512,677
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Summary for Patent: 10,512,677
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: | US16015556 |
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 active site-specific chaperone of the first composition is present in an amount from about 20 mg/mL to about 200 mg/mL, and wherein the acid α-glucosidase of the second composition is present in an amount from about 5 mg/mL to about 500 mg/mL.2. The method of claim 1 , wherein the acid α-glucosidase of the second composition is present in an amount from about 5 mg/mL to about 250 mg/mL.3. The method of claim 2 , wherein the acid α-glucosidase of the second composition is present in an amount from about 10 mg/mL to about 200 mg/mL.4. The method of claim 1 , wherein the acid α-glucosidase of the second composition is present in an amount of about 25 mg/mL.5. The method of claim 1 , wherein at least one of the first composition and the second composition is a liquid formulation.6. The method of claim 1 , wherein at least one of the first composition and the second composition further comprises an excipient.7. The method of claim 6 , wherein the excipient is selected from the group consisting of polyethylene glycol 400 claim 6 , arginine claim 6 , glutamic acid claim 6 , proline claim 6 , gammacyclodextrin claim 6 , and combinations thereof.8. The method of claim 1 , wherein at least one of the first composition and second composition further comprises a buffer.9. The method of claim 8 , wherein the buffer is selected from the group consisting of citrate buffer claim 8 , acetate buffer claim 8 , bicarbonate buffer claim 8 , phosphate buffer claim 8 , and combinations thereof.10. The method of claim 1 , wherein the active site-specific chaperone of the first composition is n-butyl-deoxynojirimycin or a pharmaceutically acceptable salt thereof.11. The method of claim 1 , wherein the active site-specific chaperone of the first composition is 1-deoxynojirimycin or a pharmaceutically acceptable salt thereof.12. The method of claim 1 , wherein at least one of the first composition and the second composition are administered independently by subcutaneous injections.13. The method of claim 12 , wherein at least one of the first composition and the second composition are administered independently with separate subcutaneous injections of 1 claim 12 , 2 claim 12 , 3 claim 12 , 4 claim 12 , 5 claim 12 , 6 claim 12 , 7 claim 12 , 8 claim 12 , 9 or 10 milliliters.14. The method of claim 1 , wherein at least one of the first composition and the second composition are administered intravenously as separate independent injections.15. The method of claim 1 , wherein the first composition is administered orally and the second composition is administered intravenously. |
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