Arrowhead Pharmaceuticals Gets FDA Nod on Orphan Drug Designation

Pasadena-based Arrowhead Pharmaceuticals said the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its ARO-ANG3.

ARO-ANG3 is investigational medicine being developed for the treatment of homozygous familial hypercholesterolemia (HoFH), a rare inherited disorder of lipoprotein metabolism.

The designation was granted by the FDA’s Office of Orphan Products Development (OOPD), which has a mission to advance the development of products that demonstrate promise for in combating diseases or conditions that affect fewer than 200,000 people in the United States.

Orphan drug designation provides incentives for sponsors to develop products for rare diseases. These incentives may include a partial tax credit for certain clinical trial expenditures, the waiver of certain FDA user fees, and potential eligibility for seven years of orphan drug marketing exclusivity.

HoFH is a life-threatening disease, characterized by elevated levels of low-density lipoprotein cholesterol (LDL-C) which can cause premature cardiovascular disease. Without adequate treatment, cholesterol buildup in the arterial wall could lead to early atherosclerosis and cardiovascular disease, even in childhood.

The disease is usually diagnosed in infancy or childhood but occasionally the diagnosis may be delayed until later in life. Diagnosis of HoFH requires a careful clinical, laboratory, and family evaluation followed by molecular genetic testing. Treatment is complex and patients should be referred to a specialized lipid unit that is experienced in the treatment of such patients.

Arrowhead’s ARO-ANG3 is a subcutaneously administered RNA interference (RNAi)-based medicine that targets Angiopoietin-like 3 or ANGPTL3 being developed for the treatment of dyslipidemias, of which HoFH is one example, and metabolic diseases. Dyslipidemia describes an abnormal amount of lipids – triglycerides, cholesterol and fat phospholipids – in the blood, often due to diet and lifestyle.

The company began dosing ARO-ANG3 in a first-in-human study in January intended to evaluate the medicine’s safety, tolerability, pharmacokinetics, and pharmacodynamic effects in adult healthy volunteers and patients with dyslipidemia. The study is designed to enroll up to 70 subjects.

Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them.

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