ARTICLE | Regulation
Inhibrx blazing regulatory trail in alpha-antitrypsin deficiency
San Diego company aims to be first to use functional AAT as a surrogate endpoint for AATD
October 20, 2022 11:38 PM UTC
Inhibrx is on the path to accelerated approval with a therapy that normalizes AAT levels and could establish functional AAT as a surrogate endpoint for alpha-1 antitrypsin deficiency, clearing the way for other companies working on similar mechanisms.
The company forecasts about $5 billion in peak annual sales — $3 billion in AATD and another $2 billion in graft-versus-host disease (GvHD), a planned follow-on indication. The company is aiming for an accelerated approval to treat AATD in 2026 and a full approval in GvHD the same year. ...