The companies expect to recruit around 50 male and female patients suffering from Fabry disease and who are presently receiving ERT treatment, and who have a genetic mutation that may be addressable with migalastat HCI.
The primary outcome of efficacy will be renal function as measured by glomerular filtration rate (GFR).
Amicus chairman and CEO John Crowley said this is an important step in their clinical development plan and builds on the latest encouraging safety and renal function data from our ongoing Phase II extension study.