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news.Amicus Therapeutics has achieved target enrollment of 56 patients in second Phase 3 fabry disease study of migalastat HCl monotherapy and expects final enrollment by the end of 2012.
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Amicus is developing the investigational pharmacological chaperone Migalastat HCl under collaboration with GlaxoSmithKline.
Study 012, designed to compare oral migalastat HCl to ERTs (Fabrazyme and Replagal), enrolled Fabry disease patients with genetic mutations amenable to migalastat HCl as a monotherapy and who were on ERT for at least a period of 12 months.
The renal function evaluated by Glomerular Filtration Rate at 18 months is the primary outcome measure of the ATTRACT, or FAB-AT1001-012 study.
Amicus chairman and chief executive officer John Crowley said the company met the enrollment objectives for the Phase 3 Fabry monotherapy study.
"The willingness of these Fabry patients to switch from an approved ERT to migalastat HCl to participate in this study highlights the unmet medical needs that persist in this community," Crowley added.
"We look forward to evaluating the effects of migalastat HCl, as well as currently used ERTs, on renal function over the course of 18 months of treatment in this study."