FDA grants orphan drug designation to dual-target CAR-T for B-cell ALL
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The FDA granted orphan drug designation to CT120, a novel chimeric antigen receptor T-cell therapy, for the treatment of B-cell acute lymphoblastic leukemia.
CT120 (IASO Biotherapeutics) is an autologous, gene-edited, fully humanized, bispecific CAR-T that targets CD19 and CD22 proteins on the surface of cancer cells.
“The orphan drug designation granted to CT120 demonstrates the FDA’s recognition and expectations for [this] product,” Wen (Maxwell) Wang, MD, PhD, CEO of IASO Biotherapeutics, said in a company-issued press release. “We look forward to the launch of this innovative therapy to cure more patients.”
The FDA based its designation on results of an ongoing phase 1 dose-escalation trial in China. The first four patients with B-cell ALL treated in the study achieved complete response to a single infusion CT120, according to data released by the manufacturer.
The company also reported no incidence of grade 3 or higher cytokine release syndrome or immune effector cell-associated neurotoxicity syndrome among patients with B-cell ALL who received the therapy.
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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