Migalastat success in Phase III (Study 012) for treatment of Fabry disease- Amicus Therapeutics

Amicus Therapeutics announced positive 18-month data from its second Phase III study (Study 012) of the oral small molecule chaperone migalastat HCl in Fabry patients with amenable mutations. Study 012 compared oral migalastat to standard-of-care enzyme replacement therapies (ERTs) for Fabry disease (Fabrazyme and Replagal). The co-primary outcome measures were the mean annualized changes in estimated glomerular filtration rate (eGFR) and measured (iohexol) GFR (mGFR) 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.

These results clearly show that migalastat is comparable to ERT in slowing the progression of Fabry disease and continues to demonstrate a favourable safety profile. With every-other-day oral administration and a differentiated mechanism of action, migalastat may offer significant advantages for patients without the need for bi-weekly infusions with ERT.