What is Fabry Disease?
Fabry disease is a serious, life-threatening rare genetic disorder. Fabry patients lack or have low levels of the lysosomal α-galactosidase-A enzyme, resulting in the progressive accumulation of abnormal deposits of a fatty substance called globotriaosylceramide, or Gb3, throughout the body. The abnormal accumulation of Gb3 increases with time, primarily in the blood vessel walls, resulting in the narrowing of channels of blood vessels and leading to decreases in both blood flow and tissue nourishment. The ultimate consequences of Gb3 deposition range from episodes of pain and impaired peripheral sensation to end-organ failure, particularly of the kidneys, but also of the heart and the cerebrovascular system. Fabry disease occurs in one per 40,000 to 60,000 males.
ABOUT Elfabrio®
Elfabrio® (pegunigalsidase alfa) is an Enzyme Replacement Therapy (or ERT), replacing the deficient α-galactosidase-A enzyme with a recombinant form of the protein by administration through intravenous infusion. Elfabrio® is indicated for the treatment of adult patients with confirmed Fabry disease. Pegunigalsidase alfa is expressed in a plant cell culture, with an addition of a unique covalently bound polyethylene glycol polymer linker. For more information, please visit www.elfabrio.com
