High concentration α-glucosidase compositions for the treatment of Pompe disease

US9303249B2 · kind B2 · utility

Assignee

• Amicus Therapeutics, Inc. · US

Inventors

• Kenneth Valenzano · East Brunswick Township, US
• John Crowley · Princeton, US
• Richie Khanna · Raritan, US
• John Flanagan · Berkeley, US

Key dates

• Filing date: Dec 13, 2013
• Grant date: Apr 5, 2016
• Expiry date: Dec 13, 2033

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12Y302/0102
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

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.