Arrowhead Pharmaceuticals Receives Breakthrough Therapy Designation from U.S. FDA for ARO-AAT for the Treatment of Alpha-1 Antitrypsin Deficiency Associated Liver Disease
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Thursday, July 29, 2021
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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.
Key Points:
- 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.
- Individuals with the homozygous PiZZ genotype have severe deficiency of functional AAT leading to pulmonary disease and liver disease.