FDA News

FDA grants orphan drug status to anti-BCMA CAR T-cell therapy for multiple myeloma

The FDA has given orphan drug status to CT053, an investigational chimeric antigen receptor T-cell therapy for the treatment of multiple myeloma.

CT053 (CARsgen Therapeutics) is an autologous, fully human CAR T-cell therapy that targets the B-cell maturation antigen on the surface of cancer cells.

CARsgen’s CT053 is one of the company’s three CAR T-cell products approved for early-stage clinical trials. The others include humanized CD19 CAR-T for B-cell leukemia and lymphoma and GPC3 CAR-T for hepatocellular carcinoma and non-small cell lung cancer.

"FDA orphan designation is an important regulatory milestone in the continued development and commercialization of CT053 anti-BCMA CAR-T cells," Zonghai Li, MD, PhD, founder, CEO and chief scientific officer of CARsgen said in a press release.

Li added that CT053 showed “outstanding potency” during an exploratory phase 1 clinical study in China, where 19 of 24 patients with relapsed or refractory multiple myeloma had a complete response to therapy. In addition, there were no cases of high-grade (3 or 4) cytokine release syndrome during the study.

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.

The FDA has given orphan drug status to CT053, an investigational chimeric antigen receptor T-cell therapy for the treatment of multiple myeloma.

CT053 (CARsgen Therapeutics) is an autologous, fully human CAR T-cell therapy that targets the B-cell maturation antigen on the surface of cancer cells.

CARsgen’s CT053 is one of the company’s three CAR T-cell products approved for early-stage clinical trials. The others include humanized CD19 CAR-T for B-cell leukemia and lymphoma and GPC3 CAR-T for hepatocellular carcinoma and non-small cell lung cancer.

"FDA orphan designation is an important regulatory milestone in the continued development and commercialization of CT053 anti-BCMA CAR-T cells," Zonghai Li, MD, PhD, founder, CEO and chief scientific officer of CARsgen said in a press release.

Li added that CT053 showed “outstanding potency” during an exploratory phase 1 clinical study in China, where 19 of 24 patients with relapsed or refractory multiple myeloma had a complete response to therapy. In addition, there were no cases of high-grade (3 or 4) cytokine release syndrome during the study.

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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