The Company expects to initiate a Phase 2 clinical trial of AT2220 for Pompe disease in early 2008. "We are pleased to be able to advance this program forward in development and explore the potential for AT2220 as a new therapeutic option for people living with Pompe disease," said John F. Crowley, President and CEO of Amicus Therapeutics.
About Pompe Disease
Pompe disease affects an estimated 5,000-10,000 patients worldwide and is clinically heterogeneous in the age of onset, the extent of organ involvement, and the rate of progression. The early onset form of the disease is the most severe, progresses most rapidly, and is characterized by musculoskeletal, pulmonary, gastrointestinal, and cardiac symptoms that usually lead to death from cardio-respiratory failure between 1 and 2 years of age. The late onset form of the disease begins between childhood and adulthood and has a slower rate of progression that is characterized by musculoskeletal and pulmonary symptoms that usually lead to progressive weakness and respiratory insufficiency. The U.S. Food and Drug Administration's Office of Orphan Products Development has granted orphan drug designation for the active ingredient in AT2220 in the United States.
About Amicus Therapeutics
Amicus Therapeutics is a biopharmaceutical company developing novel,
oral therapeutics known as pharmacological chaperones for the treatment of
a range of human genetic diseases. Pharmacological chaperone technology
involves the use of small molecules that selectively bind to and stabilize
proteins in cells, leading to improved protein folding and trafficking, and
increased activity. Amicus is initially targeting lysosomal storage
disorders, which are severe, chronic genetic diseases with unmet medical
needs. Amicus has two product candidates in Phase II clinical trials,
Amigal(TM) for the treatment of Fabry
|SOURCE Amicus Therapeutics|
Copyright©2007 PR Newswire.
All rights reserved