The FINANCIAL — GlaxoSmithKline (GSK) and Amicus Therapeutics today announced the first patient has commenced dosing in a Phase III global registration study (Study 012) to compare the safety and efficacy of Amigal™ (migalastat HCl) and enzyme replacement therapy (ERT) for the treatment of Fabry disease.
The randomised, open-label, 18-month study will provide longer-term clinical data comparing migalastat HCl to ERT in patients with Fabry disease, a rare inherited lysosomal storage disorder.
GSK and Amicus are targeting up to 50 sites globally to enrol approximately 50 male and female Fabry patients who are currently 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).
John F. Crowley, Chairman and Chief Executive Officer of Amicus, stated, "In collaboration with GSK we are pleased to announce the dosing of the first patient in Study 012, the first Phase III pivotal study to compare Amigal to ERT. This is an important step in our clinical development plan and builds on the latest encouraging safety and renal function data from our ongoing Phase II extension study."
Study 012 is the second of two Phase III studies intended to support the worldwide registration of migalastat HCl for Fabry disease. Amicus and GSK are also conducting a six-month, placebo-controlled Phase III study (Study 011) of migalastat HCI at 37 sites worldwide to support marketing applications for the U.S. Food and Drug Administration (FDA) and other regulatory agencies.
"We believe Amigal has the potential to provide an important treatment option in Fabry disease," said Dr. Philippe Monteyne, Head of Development and Chief Medical Officer for GSK Rare Diseases. "We are delighted with the progress that the joint Amicus-GSK team has made to advance the clinical development program since we entered an alliance in October 2010."
Discussion about this post