Monday, June 24, 2019
Arrowhead Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to ARO-APOC3 for the treatment of familial chylomicronemia syndrome (FCS). There is no currently available therapy in the U.S. that can adequately treat FCS.
ARO-APOC3 is a subcutaneously administered RNAi therapeutic targeting apolipoprotein C-III (APOC3) currently being developed as a potential treatment for patients with severe hypertriglyceridemia and FCS. The company began dosing ARO-APOC3 in a first-in-human study in March 2019. The trial is a Phase 1 single and multiple dose study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of ARO-APOC3 in up to 63 adult healthy volunteers with elevated triglycerides and patients with severe hypertriglyceridemia and FCS.
The mission of the FDA’s Office of Orphan Products Development (OOPD) 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 U.S. 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.