Fabry’s disease is an inherited lysosomal storage disorder brought about by the deficiency of the alpha-galactosidase A (alpha-Gal A)enzyme.
The extension study designed to evaluate the long-term safety and efficacy of migalastat HCl showed that treatment with the drug was well tolerated with no drug-related serious adverse events, with the exception of the common headache, arthralgia, diarrhea and fatigue.
Amicus, in partnership with GlaxoSmithKline (GSK) is sponsoring an ongoing Phase 2 study to evaluate the co-administration of migalastat HCl with ERT for Fabry disease, the results of which are expected in the second half of 2011.
Amicus’ lead program is in Phase 3 for the treatment of Fabry disease.
Amicus Therapeutics also plans to present its preclinical study data evaluating the co-administration of pharmacological chaperones with enzyme replacement therapy (ERT) in Fabry disease, and in genetically defined subpopulations of Parkinson’s disease and Alzheimer’s disease.