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Amicus Therapeutics Announces Additional Preliminary Results from Open Label Phase 2 Study


Amicus Therapeutics (NASDAQ: FOLD), today announced additional preliminary results from an open-label Phase 2 drug-drug interaction study (Study 013) to evaluate a single oral dose of migalastat HCl (150 mg or 450 mg) co-administered with enzyme replacement therapy (ERT) in males with Fabry disease. In a poster at the American Society of Human Genetics (ASHG) Annual Meeting Dr. David G. Warnock, University of Alabama-Birmingham, presented results from all 12 patients in the migalastat HCl 150 mg dose group.

Amicus, in collaboration with GlaxoSmithKline (GSK), is developing the investigational pharmacological chaperone migalastat HCl as a monotherapy and in combination with ERT for the treatment of Fabry disease. When co-administered with ERT, migalastat HCl binds to and stabilizes infused enzyme in the circulation.

Dr. Warnock said, "Migalastat HCl 150 mg co-administered with two different doses of Fabrazyme, as well as one dose of Replagal, appeared to increase enzyme activity compared to each of these ERTs alone. The use of a pharmacological chaperone to maintain infused alpha Gal-A enzymes in optimally active form represents a potential approach to managing ERT outcomes in patients with Fabry disease."

Results were presented for a total of 12 patients who received migalastat

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Posted-In: News Guidance Management Global


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