Immuno-Oncology Resource Center
Immuno-Oncology Resource Center
October 07, 2020
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FDA grants orphan drug designation to CAR T-cell therapy for advanced gastric cancers

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The FDA granted orphan drug designation to CT041 for the treatment of adults with relapsed or refractory gastric adenocarcinoma or gastroesophageal junction adenocarcinoma, according to the agent’s manufacturer.

CT041 (CARsgen Therapeutics) is an autologous chimeric antigen receptor T-cell therapy that targets claudin 18.2, a stomach-specific isoform of claudin 18 that is highly expressed in gastric adenocarcinoma.

FDA sign
Source: Adobe Stock.

As Healio previously reported, the FDA cleared the investigational new drug application for CT041 earlier this year.

The clearance was based on phase 1 results of a clinical trial designed to assess the safety, tolerability and pharmacokinetics of the investigational therapy. Results of the trial — presented at last year’s ASCO Annual Meeting — showed an overall response rate of 33% with an acceptable safety profile.

The phase 1B portion of an open-label, multicenter clinical trial of CT041 is currently enrolling patients, according to a CARsgen Therapeutics-issued press release. The study will evaluate the safety and efficacy of the therapy among patients with advanced gastric, gastroesophageal or pancreatic adenocarcinoma whose tumors express claudin 18.2.

“Despite the development of novel therapies, gastric cancer is still a disease with one of the highest unmet medical needs,” Zonghai Li, MD, PhD, founder, CEO and chief scientific officer of CARsgen, said in the release. “Our goal is to continue the development of novel, safe and effective immunotherapies. This is our long-standing commitment to [patients with cancer] worldwide."

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.