Amicus has initiated phase-III trial with its investigational drug, Amigal (migalastat hydrochloride), for the treatment of Fabry disease.
The company has reached an agreement with FDA on the key protocol design elements of the pivotal trial, including the use of the surrogate primary endpoint of the change in the amount of kidney interstitial capillary GL-3.
FDA said that the company is eligible to seek accelerated approval for Amigal according to Subpart H regulations. The company has begun submitting the phase-III protocol to investigational sites worldwide, and expects to begin the dosing of subjects in the second half of this year.
John Crowley, President and CEO, Amicus, said: We are very pleased with the outcome of our interactions with FDA around the design of this pivotal study and are confident we have set the stage for a successful Phase 3 study. We continue to believe that Amigal may be an important treatment option for patients who suffer with Fabry disease and a significant step forward for them and their families.