John F. Crowley, President and CEO of Amicus Therapeutics, added, "We are very pleased with this additional set of Phase 2 data and are very confident we have a solid basis for a successful Phase 3 program. We continue to work in collaboration with the FDA and remain on track to finalize our protocol and initiate the Phase 3 program in the second quarter of this year."
In January 2009, Amicus announced that the FDA supports a Phase 3 clinical trial comparing Amigal to placebo based on a surrogate primary endpoint of the change in the amount of kidney GL-3, the substrate that accumulates in the cells of Fabry patients. The Company expects to finalize the protocol and initiate Phase 3 development in the second quarter of this year.
Amicus is developing Amigal as part of a strategic collaboration with Shire Human Genetic Therapies (HGT), a business unit of Shire plc, to develop and commercialize Amicus' three lead pharmacological chaperone compounds for lysosomal storage disorders. Under the agreement, Shire received commercial rights outside of the United States. Amicus retains all U.S. rights.
About Fabry Disease
Fabry disease is a lysosomal storage disorder caused by inherited genetic mutations in the GLA gene, which result in deficient activity of the enzyme alpha-galactosidase A (a-Gal A). Deficient a-Gal A activity leads to lysosomal accumulation of globotriaosylceramide (GL-3), which is believed to cause the various symptoms of Fabry disease, including pain, kidney failure and increased risk of heart attack and stroke. Amigal is designed to selectively bind to
|SOURCE Amicus Therapeutics|
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