"We're very encouraged with the results of the ex vivo response study that suggest a majority of Pompe patients may be treatable with our pharmacological chaperone AT2220," said John F. Crowley, President and CEO of Amicus Therapeutics. "Based on these data and the safety data from the Phase 1 studies, we look forward to commencing a Phase 2 clinical trial in Pompe patients the first half of this year."
As of November 2007, AT2220 is being developed in partnership with Shire Human Genetic Therapies (HGT), a business unit of Shire plc, which is focused on genetic diseases.
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. A high majority of patients have the late onset form of the disease. 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 tre
|SOURCE Amicus Therapeutics|
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