This article is more than 5 years old. Information may no longer be current.
FDA Gives Orphan Drug Status to AUTO3 for ALL
Click Here to Manage Email Alerts
The FDA has granted orphan drug status to AUTO3, a cell-based immunotherapy for the treatment of acute lymphoblastic leukemia.
AUTO 3 (Autolus Therapeutics) is a programmed T-cell therapy containing two independent chimeric antigen receptors targeting CD19 and CD22 that are independently optimized for single-target activity.
“By simultaneously targeting two B cell antigens, AUTO3 is designed to minimize relapse due to single antigen loss in patients with B cell malignancies,” according to a press release from the manufacturer.
“We believe that AUTO3 has the potential to be a best-in-class therapy in pediatric ALL by addressing antigen escape, a common cause of relapse in these patients,” Christian Itin, PhD, CEO of Autolus, said in a press release. “AUTO3 may also provide an improved safety profile over currently marketed CAR T therapies with low levels of severe [cytokine release syndrome] and neurotoxicity observed in clinical studies.”
AUTO3 is currently being tested in pediatric patients with ALL as part of the AMELIA clinical trial and in patients with diffuse large B cell lymphoma in the ALEXANDER clinical trial.
Data from the American Cancer Society suggest there will be 5,930 new cases of ALL and approximately 1,500 deaths in 2019. Sixty percent of cases will be in patients aged younger than 20 years.
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.