The Food and Drug Administration (FDA) has granted Fast Track designation to ARO-AAT (Arrowhead Pharmaceuticals Inc), an investigational treatment for a rare genetic liver disease associated with alpha-1 antitrypsin deficiency (AATD). 

ARO-AAT, a subcutaneously administered RNAi therapeutic, works by reducing production of the inflammatory mutant Z-AAT protein to stop the progression of liver disease in AATD patients. The Company will be initiating the phase 2/3 SEQUOIA study in the US and Europe and a phase 2 open-label study in Europe. 

The SEQUOIA trial will evaluate the safety, tolerability and effect on liver histologic parameters following administration of ARO-AAT in 120 patients with AATD-associated liver disease. In the phase 2 AROAAT2002 study, the effect of ARO-AAT on a histological liver disease activity scale will be evaluated in patients with AAT-associated liver disease over time.

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“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, MD, 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.”

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