Migalastat (Galafold™)-a small molecule drug developed by Amicus Therapeutics 
that restores the activity of specific mutant forms of α-galactosidase-has been 
approved for the treatment of Fabry disease in the EU in patients with amenable 
mutations. Fabry disease is a rare disorder that results in a deficiency or 
absence of α-galactosidase, leading to accumulation of globotriaosylceramide in 
the lysosomes of various cells. This article summarizes the milestones in the 
development of migalastat leading to this first approval in the EU for the 
long-term treatment of adults and adolescents aged ≥16 years with a confirmed 
diagnosis of Fabry disease.