Pharma Focus Asia

Arrowhead Pharmaceuticals Receives Orphan Drug Designation for ARO-AAT

Friday, February 16, 2018

Arrowhead Pharmaceuticals Inc. announced the United States Food and Drug Administration (FDA) has granted orphan drug designation to ARO-AAT, Arrowhead’s second-generation investigational medicine for the treatment of a rare genetic liver disease associated with alpha-1 antitrypsin deficiency. Arrowhead filed a Clinical Trial Application in December 2017 requesting regulatory permission to begin first-in-human studies of ARO-AAT.

“The Orphan Drug Act provides important incentives for sponsors to develop drugs that treat rare diseases and we look forward to more engagement with the FDA as the development of ARO-AAT progresses,” said Bruce D. Given, M.D., Arrowhead’s chief operating officer and head of R&D.

The FDA Office of Orphan Products Development (OOPD) mission is to advance the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions. In fulfilling that task, 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.

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