Arrowhead Pharmaceuticals Receives Orphan Drug Designation for ARO-AAT

Home/Newsroom/

Arrowhead Pharmaceuticals Receives...

PASADENA, Calif.–(BUSINESS WIRE)–

Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today 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.

For more information, please visit www.arrowheadpharma.com,

or follow us on Twitter @ArrowheadPharma.

To be added to the Company’s email list and receive news directly,

please visit http://ir.arrowheadpharma.com/alerts.cfm.

Safe Harbor Statement under the Private Securities Litigation Reform

Act:

This news release contains forward-looking statements within the

meaning of the “safe harbor” provisions of the Private Securities

Litigation Reform Act of 1995. These statements are based upon our

current expectations and speak only as of the date hereof. Our actual

results may differ materially and adversely from those expressed in any

forward-looking statements as a result of various factors and

uncertainties, including the safety and efficacy of our product

candidates, the duration and impact of regulatory delays in our clinical

programs, our ability to finance our operations, the future success of

our scientific studies, our ability to successfully develop drug

candidates, the timing for starting and completing clinical trials,

rapid technological change in our markets, and the enforcement of our

intellectual property rights. Our most recent Annual Report on Form 10-K

and subsequent Quarterly Reports on Form 10-Q discuss some of the

important risk factors that may affect our business, results of

operations and financial condition. We assume no obligation to update or

revise forward-looking statements to reflect new events or circumstances.

Source: Arrowhead Pharmaceuticals, Inc.

Arrowhead Pharmaceuticals, Inc.
Vince Anzalone, CFA
626-304-3400
ir@arrowheadpharma.com
or
Investors

and Media:
LifeSci Advisors, LLC
Brian Ritchie
212-915-2578
britchie@lifesciadvisors.com
www.lifesciadvisors.com

Source: Arrowhead Pharmaceuticals, Inc.

News Provided by Acquire Media