Research & Development
Arrowhead Pharmaceuticals Receives Orphan Drug Designation for ARO-ANG3
17 July 2019 - - The US Food and Drug Administration has granted orphan drug designation to ARO-ANG3 for the treatment of homozygous familial hypercholesterolemia, US-based Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) said.
ARO-ANG3 is a subcutaneously administered RNA interference -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 January 2019.
AROANG1001 (NCT03747224) is a Phase 1 single and multiple dose study 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.
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 Office of Orphan Products Development is to advance the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions that affect fewer than 200,000 people in the US.
In fulfilling that task, the OOPD evaluates scientific and clinical data submissions from sponsors to identify and designate products as promising for rare diseases and to further advance scientific development of such promising medical products.
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, if approved.
Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them.
Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes.
RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead's RNAi-based therapeutics leverage this natural pathway of gene silencing.