Migalastat: Additional Phase III data

Data from paired biopsies of 8 treatment-naive Fabry’s disease patients with amenable genetic mutations in the double-blind, placebo-controlled, international Phase III FACETS (Study 011) trial showed that 150 mg oral migalastat every other day significantly reduced GL-3 levels in podocytes at 6 months compared to baseline (p=0.02). Amenable mutations were defined as having an absolute increase of 3% of wild-type alpha galactosidase A activity and a relative increase of 20% when exposed to migalastat in a cell-based in vitro assay. Data were presented at WORLDSymposium in San Diego. Amicus has previously reported data from FACETS and its open-label Phase III extension Study 041 (see BioCentury, Dec. 24, 2012; Feb. 18, 2013; May 5, 2014; Jan. 19, 2015 & Feb. 16, 2015). ...