Arrowhead Pharmaceuticals receives breakthrough therapy designation from U.S. FDA for ARO-AAT for the treatment of alfa-1 anti-trypsin deficiency associated liver disease

Arrowhead Pharmaceuticals

29 July 2021 - Full enrollment reached in Phase 2 SEQUOIA study of ARO-AAT.

Arrowhead Pharmaceuticals today announced that it was granted breakthrough therapy designation by the U.S. FDA for ARO-AAT, also known as TAK-999, the company’s second-generation investigational RNA interference therapeutic being co-developed with Takeda as a treatment for the rare genetic liver disease associated with alfa-1 anti-trypsin deficiency. 

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.

Read Arrowhead Pharmaceuticals press release

Michael Wonder

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Michael Wonder