The four open-label, multi-national Phase 2 trials of Amigal enrolled 18 men and 9 women with Fabry disease between the ages of 17 and 65. The four studies examined various dose levels and frequencies of Amigal administration and had 12 or 24 week primary treatment arms with an optional treatment extension.
Twenty-six patients completed the primary treatment arms and all entered the optional treatment extension. The 26 patients had 21 different missense genetic mutations that cause Fabry disease. The mutations represented the full spectrum of Fabry patients, including those with both early-onset and late- onset forms of the disease. Twenty-three patients are currently being treated with Amigal under the treatment extension, including 8 who have been treated for more than a year and 4 who have been treated for almost 2 years.
"The positive results of these first trials of a pharmacological
chaperone in Fabry disease are impressive," said Raphael Schiffmann, M.D.,
Lead Investigator at the Metabolic Neurology Branch of the National
Institute of Neurological Disorders and Stroke (NINDS), a part of the
National Institutes of Health, and a principal investigator in one of the
Amigal clinical trials. "I believe this technology has the potential to be
an important new treatment option for many Fabry disease patients."
The key findings in the Phase 2 studies were:
* Amigal was generally safe and well-tolerated at all doses evaluated. No
drug-related serious adverse events were reported during the primary
|SOURCE Amicus Therapeutics Inc.|
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