treatment arm, and none have been reported during the treatment
* Twenty-four out of 26 patients demonstrated an increase in a-GAL as
measured in white blood cells, kidney, and skin.
* a-GAL increases were seen in patients with both low levels of residual
enzyme activity (<3%) at baseline as well as patients presenting with
higher baseline levels (greater than or equal to 3%).
* Kidney GL-3 levels as measured in urine or biopsies were decreased in
patients who demonstrated greater increases in levels of a-GAL.
* Renal and cardiac function results were encouraging, including those
seen in patients treated for nearly two years.
* Patient responses were consistent with the results of in vitro testing
of Fabry mutations, thus improving the ability to select likely
responders for future studies.
"These data demonstrate that Amigal has a meaningful effect on a range of genetic mutations in Fabry disease and thus has the potential to treat a significant portion of the Fabry patient population," said William Wilcox, M.D., Ph.D., Director of the Metabolic Disorders Clinic at Cedars-Sinai Medical Center and a principal investigator in one of the Amigal clinical trials.
Amicus expects that the results will be presented again at the American College of Medical Genetics (ACMG) Annual Meeting on March 12-16, 2008, in Phoenix, Arizona.
Based on the results of these Phase 2 trials, Amicus and Shire plan to meet with US and European regulatory authorities to discuss the design of a Phase 3 clinical trial for Amigal.
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). Deficient a-GAL activity leads to
lysosomal accumulation of globotriaosylceramide (GL-3), which is believed
to cause the various s
|SOURCE Amicus Therapeutics Inc.|
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