The U.S. Food and Drug Administration (FDA) granted orphan drug designation to ARO-ANG3 (Arrowhead Pharmaceuticals) for the treatment of homozygous familial hypercholesterolemia (HoFH).
ARO-ANG3 is a RNA interference (RNAi)-based medicine that targets angiopoietin like protein 3 (ANGPTL3). The drug is being developed for the treatment of various dyslipidemias, including HoFH.
Earlier this year, the company began a Phase 1 dosing study (NCT03747224) to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of ARO-ANG3 in adult healthy volunteers and patients with dyslipidemia. The study is designed to enroll up to 70 subjects. subcutaneously administered
HoFH is a rare genetic disorder involving lipoprotein metabolism. It is characterized by a dramatic elevation of low-density lipoprotein cholesterol (LDL-C) that will lead to premature cardiovascular disease. Without proper treatment, cholesterol buildup leads to early atherosclerosis and cardiovascular disease, even in childhood1.
FDA’s Office of Orphan Products Development (OOPD) advances the evaluation and development of products that show promise for diagnosing and/or treating rare diseases. A rare disease is defined as one that affects fewer than 200,000 people in the U.S. An orphan drug designation provides sponsors with various incentives to develop products for rare diseases. These incentives may include a partial tax credit for clinical trial expenditures, the waiver of FDA user fees, and potential eligibility for seven years of orphan drug marketing exclusivity (if the drug gets approved by the FDA).