Stable parenteral DNJ compositions

US9827189B2 · kind B2 · utility

Assignee

• Amicus Therapeutics, Inc. · US

Inventors

• Hing Char · East Brunswick Township, US
• Sergey Tesler · Sparta, US
• Jiping Yang · San Jose, US
• Enrique Diloné · Jalan Pedongkelan, US

Key dates

• Filing date: Jan 9, 2014
• Grant date: Nov 28, 2017
• Expiry date: Jan 9, 2034

Classification

• Technology area (CPC A): Human Necessities
• CPC primary: A61P43/00
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

A stable pharmaceutical composition that includes an active agent selected from 1-deoxynojirimycin, a pharmaceutically acceptable salt thereof, or a derivative thereof, and a buffer, wherein the stable pharmaceutical composition is capable of being parenterally administered to a human without deleterious health effects. Pompe disease is an example of a lysosomal storage disorder. Pompe disease is caused by a deficiency in the enzyme acid alpha-glucosidase (GAA). GAA metabolizes glycogen, a storage form of sugar used for energy, into glucose.