Rare Daily Staff

The U.S. Food and Drug Administration has granted Fast Track designation to Arrowhead Pharmaceuticals’ ARO-AAT, the company’s second generation subcutaneously administered RNA interference therapy as a treatment for a rare genetic liver disease associated with alpha-1 antitrypsin deficiency.

Alpha-1 antitrypsin deficiency (AATD) is a rare genetic disease that results in a lack of the protein AAT and can cause lung disease and liver disease as a result. Arrowhead is in final preparations to initiate SEQUOIA, a potentially pivotal phase 2/3 clinical study in the United States and Europe, and AROAAT2002, a phase 2 open-label clinical study in Europe.

“With no currently approved agents to treat AATD-associated liver disease, alpha-1 patients and their physicians have an urgent need for new therapeutic options,” said Bruce Given, Arrowhead’s chief operating officer and head of R&D. “We view this FDA Fast Track designation as continued support that ARO-AAT has the potential to address this unmet need. Importantly, Fast Track designation provides a number of important advantages that could expedite the development and review of ARO-AAT.”

Fast Track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to the patient earlier.

A drug that receives fast track designation is eligible for more frequent meetings and communications with FDA to discuss the drug’s development plan and ensure collection of appropriate data needed to support drug approval, eligibility for accelerated approval and priority review, and the opportunity to submit completed sections of its New Drug Application for review by FDA in a rolling process.

Photo: Bruce Given, Arrowhead’s chief operating officer and head of R&D

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