Arrowhead gains Breakthrough designation for lead candidate

29 July 2021

US biotech Arrowhead Pharmaceuticals (Nasdaq: ARWR) today announced that it was granted Breakthrough Therapy designation (BTD) by the US Food and Drug Administration for ARO-AAT, also known as TAK-999, the company’s second-generation investigational RNA interference (RNAi) therapeutic.

The compound is being co-developed with Japan’s Takeda Pharmaceutical (TYO: 4502) 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.

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