Arrowhead Receives Positive EMA Opinion on Orphan Designation for ARO-AAT for Treatment of Congenital Alpha-1 Antitrypsin Deficiency

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Arrowhead Receives Positive EMA...

PASADENA, Calif.–(BUSINESS WIRE)–Jun. 27, 2018–
Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that the
European Medicines Agency (EMA) Committee for Orphan Medicinal Products
(COMP) issued a positive opinion on Arrowhead’s application for orphan
designation of its RNAi candidate, ARO-AAT, for the treatment of
congenital alpha-1 antitrypsin deficiency. ARO-AAT was previously
granted orphan drug designation by the United States Food and Drug
Administration in February 2018.

Bruce Given, M.D., Arrowhead’s chief operating officer and head of R&D,
said, “ARO-AAT has the potential to provide patients and physicians with
a much-needed treatment option for liver disease associated with alpha-1
antitrypsin deficiency, a rare genetic disorder. The COMP’s adoption of
a positive opinion for ARO-AAT orphan drug designation is another
important milestone for this program, which continues to advance
rapidly.”

The positive opinion issued by COMP will be sent to the European
Commission which is expected to grant the orphan designation within 30
days. Orphan designation in the EU allows Arrowhead to benefit from a
number of key incentives, including reduced regulatory fees, protocol
assistance, and market exclusivity, to develop a medicine for the
treatment of a rare disease affecting not more than five in 10,000
people in the European Union.

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,
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This news release contains forward-looking statements within the
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Litigation Reform Act of 1995. These statements are based upon our
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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
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and subsequent Quarterly Reports on Form 10-Q discuss some of the
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Source: Arrowhead Pharmaceuticals, Inc.

Source: Arrowhead Pharmaceuticals Inc.

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