Arrowhead Gets Breakthrough Tag for Treatment of Alpha-1 Antitrypsin Deficiency Associated Liver Diseas

 Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that it was granted Breakthrough Therapy designation (BTD) by the U.S. Food and Drug Administration (FDA) for ARO-AAT, also known as TAK-999, the company’s second-generation investigational RNA interference (RNAi) therapeutic being co-developed with Takeda Pharmaceutical Company Limited ("Takeda”) as a treatment for the rare genetic liver disease associated with alpha-1 antitrypsin deficiency (AATD). Investigational therapy ARO-AAT was also previously granted Orphan Drug designation and Fast Track designation from the FDA, and Orphan designation from the European Commission.

https://www.businesswire.com/news/home/20210729005199/en/Arrowhead-Pharmaceuticals-Receives-Breakthrough-Therapy-Designation-from-U.S.-FDA-for-ARO-AAT-for-the-Treatment-of-Alpha-1-Antitrypsin-Deficiency-Associated-Liver-Disease