Amicus Therapeutics reports positive Phase 3 data from Fabry study Amicus Therapeutics announced positive 18-month data from its second Phase 3 study of the oral small molecule chaperone migalastat HCl - "migalastat" - in Fabry patients with amenable mutations. Study 012 compared oral migalastat to standard-of-care enzyme replacement therapies for Fabry disease. The co-primary outcome measures were the mean annualized changes in estimated glomerular filtration rate and measured GFR assessed by descriptive comparisons of migalastat and ERT over 18 months. Migalastat had a comparable effect to ERT on patients' kidney function as measured by the change in eGFR and mGFR. Levels of plasma lyso-Gb3, an important biomarker of disease, remained low and stable in patients with amenable mutations who switched from ERT to migalastat. Migalastat was generally safe and well-tolerated.