Arrowhead Pharmaceuticals Receives Orphan Drug Designation for ARO-ANG3
ARO-ANG3 is a subcutaneously administered RNA interference (RNAi)-based investigational medicine targeting angiopoietin like protein 3 (ANGPTL3) being developed for the treatment of dyslipidemias, such as HoFH, and metabolic diseases. The company began dosing ARO-ANG3 in a first-in-human study (AROANG1001) in
HoFH is a rare inherited disorder of lipoprotein metabolism. It is a life-threatening disease, characterized by marked elevation of low-density lipoprotein cholesterol (LDL-C) leading to premature cardiovascular disease. Without adequate treatment, cholesterol buildup in the arterial wall leads to early atherosclerosis and cardiovascular disease, even in childhood1.
The mission of the FDA’s
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1 Najam O, Ray KK. Familial hypercholesterolemia: a review of the natural history, diagnosis, and management. Cardiol Ther. 2015;4(1):25-38
Arrowhead Pharmaceuticals, Inc.
Vince Anzalone, CFA