FDA grants fast track to alpha-1 antitrypsin deficiency-related liver disease drug

The FDA granted fast track designation to ARO-AAT, Arrowhead Pharmaceuticals’ second generation subcutaneously administered RNA interference therapeutic, for the treatment of alpha-1 antitrypsin deficiency-related liver disease, according to a press release.

Arrowhead also announced that the company is in final preparations to initiate SEQUOIA (AROAAT2001), a phase 2/3 clinical study in the U.S. 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,” Bruce Given, MD, Arrowhead’s chief operating officer and head of research and development, said in the release. “We view this FDA fast track designation as continued support that ARO-AAT has the potential to address this unmet need.”

The randomized control SEQUOIA study will comprise 120 patients who will receive ARO-AAT on day 1, day 29, and approximately every 12 weeks thereafter to determine safety, pharmacodynamic dose response, and efficacy during the full two-part study.

The AROAAT2002 study will comprise up to 12 patients who will receive the same dosing schedule to evaluate multiple secondary and exploratory objectives regarding treatment outcomes.

Reference: www.arrowheadpharma.com