FDA grants orphan drug status to CAR-T for T-cell acute lymphoblastic leukemia
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The FDA granted orphan drug designation to WU-CART-007 for patients with relapsed or refractory T-cell acute lymphoblastic leukemia.
WU-CART-007 (Wugen) is an allogeneic, gene-edited chimeric antigen receptor T-cell therapy that targets the protein CD7 on the surface of cancer cells.
The therapy, derived from healthy donor cells, uses CRISPR/Cas9 gene editing to knock out CD7 and T-cell receptor alpha constant to prevent CAR T-cell fratricide and graft-versus-host disease.
The investigational agent is the first allogeneic CAR-T for T-cell malignancies to be approved for clinical testing in the United States, according to a press release issued by the manufacturer.
WU-CART-007 is being evaluated as part of a phase 1/phase 2 dose-escalation study of patients with relapsed or refractory T-cell ALL or T-cell lymphoblastic lymphoma. The international, multicenter trial is designed to assess the safety and efficacy of preconditioning lymphodepletion followed by a single dose of WU-CART-007.
Approximately 11,000 people worldwide are diagnosed with T-cell ALL or T-cell lymphoblastic lymphoma each year, according to Armin Ghobadi, MD, associate professor of medicine in the division of medical oncology at Washington University School of Medicine in St. Louis and one of the study’s principal investigators.
“About half of these patients are either resistant to frontline therapy or relapse even after initial remission and are ultimately left with very limited treatment options” he said in the release. “We look forward to advancing WU-CART-007, which has the potential to transform the care paradigm for these patients.”
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.
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