High Concentration Alpha-Glucosidase Compositions for the Treatment of Pompe Disease
AssigneeAmicus Therapeutics, Inc.
InventorsKenneth Valenzano, John Crowley, Richie Khanna, John Flanagan
Life of this patent
Estimating the term…
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.
Claims60
A composition comprising acid α-glucosidase and an active site-specific chaperone selected from the group consisting of 1-deoxynojirimycin and n-butyldeoxynojirimycin, wherein the acid α-glucosidase is present in an amount greater than about 5 mg/mL.
A method of treating Pompe disease in a subject comprising administering to the subject a composition comprising acid α-glucosidase and an active site-specific chaperone selected from the group consisting of 1-deoxynojirimycin and n-butyldeoxynojirimycin, wherein the acid α-glucosidase is present in an amount greater than about 5 mg/mL.
A method for increasing the stability of acid α-glucosidase in a formulation for subcutaneous administration to a subject, comprising combining the acid α-glucosidase with an active site-specific chaperone selected from the group consisting of 1-deoxynojirimycin and n-butyl-deoxynojirimycin, wherein the acid α-glucosidase is present in an amount greater than about 5 mg/mL.
A method of treating a tissue or an organ affected by Pompe disease in a subject comprising subcutaneous administration to the subject a composition comprising acid α-glucosidase and an active site-specific chaperone selected from the group consisting of 1-deoxynojirimycin and n-butyldeoxynojirimycin, wherein the acid α-glucosidase is present in an amount greater than about 5 mg/mL, and wherein the composition is administered in close proximity to the affected tissue or the organ.
Dependencies are inferred from the claim text.