FDA grants orphan drug designation to CAR T-cell therapy for gastric cancer
The FDA granted orphan drug designation to CTB001, an investigational chimeric antigen receptor T-cell therapy for treatment of certain gastric cancers.
CTB001 (Nanjing Bioheng Biotech) is an autologous, gene-edited CAR T-cell therapy that targets the claudin18.2 protein on the surface of cancer cells.
Claudin18.2 is a stomach-specific isoform of claudin18 that is highly expressed by several primary tumor types and metastases, including gastric cancer, pancreatic cancer, cholangiocarcinoma, ovarian cancer and lung adenocarcinoma.
The manufacturer developed the investigational cell therapy using Bioheng Explored CAR-T platform technology to enhance the product’s antitumor efficacy. Exploratory clinical studies have demonstrated favorable efficacy and safety, according to a company-issued press release.
“CTB001 is the first clinical validation of Bioheng Explored CAR-T platform technology,” the company said in a statement. “It is a very promising immunotherapy that can accurately, quickly and efficiently bring good benefits to [patients with] claudin18.2-positive cancer.”
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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