Arrowhead Pharmaceuticals Inc. (ARWR) Receives Breakthrough Therapy Designation from U.S. FDA for ARO-AAT for the Treatment of Alpha-1 Antitrypsin Deficiency Associated Liver Disease

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.

Javier San Martin, M.D., chief medical officer at Arrowhead, said: “Patients with AATD associated liver disease currently have no available treatment options other than a liver transplant. Being granted Breakthrough Therapy designation from the FDA is an important milestone for the investigational ARO-AAT program. Arrowhead and our collaborators at Takeda share a commitment to patients with AATD. We intend to utilize the benefits that BTD provides, including enhanced access to and guidance from senior management and experienced review staff at the FDA, to expedite the development of ARO-AAT. We hope to get this important investigational medicine to patients rapidly.

Dr. San Martin continued: “I am also pleased to announce that the ARO-AAT Phase 2 SEQUOIA study has recently reached full enrollment of 40 patients. The interim results we recently presented at the 2021 European Association for the Study of the Liver (EASL) International Liver Congress from the AROAAT2002 Phase 2 open label study were highly encouraging. We believe these interim results suggest that investigational ARO-AAT consistently reduced the production of the toxic mutant Z-AAT protein, which has been identified as the cause of progressive liver disease in patients with AATD. Further, these reductions over 6 and 12 months led to multiple important signals associated with healing of liver disease in patients with fibrosis due to AATD.”

BTD is a process designed to expedite the development and review of drugs that are intended to treat a serious life-threatening disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. A drug that receives BTD is eligible for all Fast Track designation features, intensive FDA guidance on an efficient drug development program, and organizational commitment involving senior managers at the FDA.1

You May Also Be Interested In

• AstraZeneca (AZN) Announces Voydeya Approval in EU as add-on to ravulizumab or eculizumab
• BeiGene (BGNE) Receives EU Approval for Tislelizumab
• Adicet Bio (ACET) Highlights Preclinical Data Supporting IND Readiness for ADI-270 in an Oral Presentation