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Arrowhead Pharmaceuticals Receives Orphan Drug Designation for ARO-AAT

3.1 from 16 votes
Friday, February 16, 2018

PASADENA, Calif.- Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR)  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.

About Arrowhead Pharmaceuticals
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.

Arrowhead Pharmaceuticals, Inc.
Vince Anzalone, CFA
Phone: 626-304-3400

Source: Arrowhead Pharmaceuticals, Inc.
3.1 from 16 votes
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