CRANBURY, N.J., Feb. 16, 2011 /PRNewswire/ -- Amicus Therapeutics (Nasdaq: FOLD) today announced that additional positive data from the ongoing Phase 2 extension study of its investigational drug Amigal™ (migalastat HCl) for Fabry disease will be presented at the Lysosomal Disease Network WORLD Symposium in Las Vegas, Nevada, February 16-18th, 2011. In addition, the Company announced that it will present encouraging data from its preclinical studies evaluating the co-administration of pharmacological chaperones with enzyme replacement therapy (ERT) in Fabry disease, as well as from preclinical studies examining the use of pharmacological chaperones for the treatment of genetically defined subpopulations of Parkinson's disease and Alzheimer's disease.
Preliminary Data Update from Phase 2 Long-Term Extension Study of Migalastat HCl for Fabry DiseaseTwenty-six subjects completed either 12 or 24 weeks of treatment with migalastat HCl during the initial Phase 2 studies and 23 subjects enrolled in a separate, long-term extension study designed to evaluate the long-term safety and efficacy of migalastat HCl. Over the course of the initial Phase 2 and extension studies, 15 subjects have been treated with migalastat HCl for more than 3 years and 7 subjects have been treated with migalastat HCl for more than 4 years. Seventeen subjects continue to receive treatment in the ongoing extension study.
During the course of the extension study, treatment with migalastat HCl has continued to be generally well tolerated, with no drug-related serious adverse events. The most common adverse events have been headache, arthralgia, diarrhea and fatigue.
Renal function continues to be evaluated by two measures in the extension study, estimated glomerular filtration rate (eGFR) and 24-hour urine protein. Preliminary data indicate that eGFR has remained stable out to 3-4 years
|SOURCE Amicus Therapeutics|
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