Arrowhead Research

Arrowhead receives Orphan designation for liver disease treatment ARC-AAT

Thursday, June 11, 2015

The FDA has granted Arrowhead Research’s ARC-AAT Orphan Drug designation. ARC-AAT is Arrowhead’s RNAi-based therapeutic candidate being investigated for the treatment of liver disease associated with Alpha-1 Antitrypsin Deficiency (AATD), a rare genetic disease that severely damages the liver and lungs of affected children and adults. Arrowhead currently is conducting part B of a phase I study of ARC-AAT in patients with PiZZ genotype AATD.

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