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January 21, 2022
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FDA grants orphan drug designation to CAR T-cell therapy for advanced ALL

Source:

Press Release

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The FDA granted orphan drug designation to CNCT19, a chimeric antigen receptor T-cell therapy for the treatment of relapsed or refractory acute lymphoblastic leukemia.

CNCT19 (CASI Pharmaceuticals and Juventas Cell Therapy) is an autologous, gene-edited CAR T-cell therapy that targets the CD19 protein on the surface of cancer cells.

Phase 1 study outcomes.
Data derived from Liu W, et al. Abstract 2349. Presented at: ASH Annual Meeting and Exposition; Dec. 11-14, 2021; Atlanta.

China-based CASI has partnered with Juventas Cell Therapy on a worldwide co-commercialization agreement for CNCT19. Juventas will develop the therapy and conduct related clinical trials.

If the FDA approves CNCT19, it would be the first CAR T-cell therapy developed in China available for commercial use in the U.S.

“Our partner Juventas continues to make encouraging progress in developing their CD19 CAR-T therapy,” Wei-Wu He, PhD, chairman and CEO of CASI, said in a company-issued press release. “The orphan drug designation from the FDA and the breakthrough designation status granted by the China Center of Drug Evaluation in December 2020 represent significant milestones that demonstrate our belief that CNCT19’s commercialization will not only be successful in China, but potentially on a global scale.”

A phase 2 study is underway in China to evaluate CNCT19 for adults with relapsed or refractory ALL.

At this year’s ASH Annual Meeting and Exposition, researchers presented data from 63 patients who received a single infusion of CNCT19 during the phase 1 portion of the study and had a minimum of at least 12 months of follow-up. Results showed an overall response rate of 93.8% among adults in the study, and 90.6% of adults achieved a minimal residual disease-negative response.

The majority of adults in the study achieved 6-month OS (89.6%) and 12-month OS (62.3%).

Most patients (85.7%) experienced cytokine release syndrome (grade 3, 19%). Immune effector cell-associated neurotoxicity syndrome occurred in 34.9% of patients (grade 3, 20.6%)

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.

Reference:

Wang Y, et al. Abstract 2811. Presented at: ASH Annual Meeting and Exposition; Dec. 11-14, 2021; Atlanta.