Alpha-Galactosidase A And 1-Deoxygalactonojirimycin Co-Formulation
AssigneeAmicus Therapeutics, Inc.
InventorsRichie Khanna, Kenneth Valenzano, Susan Elizabeth Fowles
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Abstract
The present application provides for compositions comprising α-galactosidase A in combination with an active site-specific chaperone for the α-galactosidase A, and methods for treating Fabry 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 α-galactosidase A enzyme formulation.
Claims20
A method of treating Fabry disease, the method comprising administering a pharmaceutical composition to a patient in need thereof, wherein the pharmaceutical composition comprises:
- α-galactosidase A at a concentration in the range of about 0.5 to about 20 μM;
- and
- 1-deoxygalactonojirimycin or a pharmaceutically acceptable salt thereof at a concentration in the range of about 50 μM to about 20,000 μM.
A method of treating Fabry disease, the method comprising intravenously administering a pharmaceutical composition to a human patient in need thereof, wherein the pharmaceutical composition comprises:
- α-galactosidase A at a concentration in the range of 0.5 and 20 μM;
- and
- 1-deoxygalactonojirimycin or a pharmaceutically acceptable salt thereof at a concentration in the range of 50 to 20,000 μM,
- wherein the pharmaceutical composition is administered as a dosage, wherein the dosage of α-galactosidase A is in the range of about 0.1 to about 5 mg/kg, and the dosage of 1-deoxygalactonojirimycin or salt thereof is on the range of about 0.1 to about 15 mg/kg.
Dependencies are inferred from the claim text.