- Fabry is a very rare lysosomal storage disorder. Patients with Fabry disease don’t produce enough of the enzyme alpha-galactosidase (GAL), which is needed to break down target complex fatty molecules.
- Fabry disease is rare and genetic. According to the Fabry Community website, 1 in 117,000 people are affected.
- As certain complex molecules build up in the kidneys, Fabry disease patients face escalating problems, including heart, brain, gastrointestinal system, and skin issues. It is also very painful.
The goal of treating Fabry disease is to help the kidneys clear globotriaosylceramide (GL-3), a complex fatty molecule. The current standard of care has been enzyme replacement therapy (ERT), mainly with Fabrazyme and Replagel. In a phase III study of 60 patients who had previously been treated with ERT, migalastat resulted in improvements in estimated and measured glomerular filtration rate (GFR).
“These results clearly show that migalastat is comparable to ERT in slowing the progression of Fabry disease and continue to demonstrate a favorable safety profile,” said Amicus CEO John Crowley.