cohort dosed least frequently.
-- As expected in this short term study, the levels of relevant markers
of Gaucher disease including platelet counts, hemoglobin levels,
glucocerebroside (substrate) levels, chitotriosidase activity and
pulmonary activation-related chemokine (PARC) levels were maintained.
"These data give us great confidence in moving our Gaucher program forward," said John F. Crowley, President and CEO of Amicus Therapeutics. "In addition to a 6-month Phase 2 study in individuals naive to ERT, which is currently underway, we plan to initiate a longer-term study in individuals switching from enzyme replacement therapy to Plicera in the second half of this year."
As of November 2007, Plicera is being developed in partnership with Shire Human Genetic Therapies (HGT), a business unit of Shire plc, which is focused on genetic diseases.
About Gaucher Disease
Gaucher disease is a lysosomal storage disorder caused by inherited genetic mutations in the GBA gene, which result in deficient activity of the enzyme acid beta-glucosidase, also known as glucocerebrosidase (GCase). Deficient GCase activity leads to lysosomal accumulation of glucocerebroside inside certain cells, which is believed to cause the various symptoms of Gaucher disease, including an enlarged liver and spleen, abnormally low levels of red blood cells and platelets and skeletal complications. In some cases there is significant impairment of the central nervous system.
Gaucher disease is estimated to affect approximately 10,000 people in the developed world. The U.S. Food and Drug Administration's Office of Orphan Products Development has granted orphan drug designation for the active ingredient in Plicera in the United States and the European Commission has designated Plicera as an orphan medicinal product in the European Union.
About Amicus Therapeutics
Amicus Therapeutics is a biopharm
|SOURCE Amicus Therapeutics|
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