Migalastat

Migalastat is prescribed to treat Fabry disease in adult patients. Fabry disease is a rare, genetic and an inherited disorder that occurs due to an accumulation or bulid-up of a type of fat known as globotriaosylceramide (GL-3) in the blood vessels, nerves, heart, the kidneys, and other organs. Migalastat is used only after the confirmation of an alpha-galactosidase enzyme deficiency or a permanent gene alteration in the alpha-galactosidase gene A (GLA) located on the X chromosome. Patients with Fabry disease may develop abnormal heart rhythm, enlargement of the heart, progressive kidney disease, stroke and early death. Migalastat works by increasing the activity of the body’s deficient enzyme rather than acting as an enzyme replacement therapy. Patients treated with migalastat over six months showed a more significant reduction in globotriaosylceramide (GL-3) in the blood vessels of the kidneys.