FDA grants orphan drug designation to humanized CAR-T for advanced multiple myeloma
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The FDA granted orphan drug designation to CT103A, a chimeric antigen receptor T-cell therapy in development for adults with relapsed or refractory multiple myeloma.
CT103A (IASO Biotherapeutics/Innovent Biologics) is an autologous, gene-edited CAR T-cell therapy that targets the B-cell maturation antigen on the surface of cancer cells.
The agent contains a unique fully human single-chain variable fragment sequence and a 4-1BB costimulatory and CD3-zeta domain encoded within the lentiviral vector.
“[The granting of orphan drug designation] to CT103A is of great significance to patients with multiple myeloma and represents the FDA’s recognition of CT103A and the clinical data provided by IASO Bio,” Wen Wang, MD, PhD, CEO and chief medical officer of IASO Bio, said in a company-issued press release. “We are looking forward to the launch of CT103A both in China and the US as soon as possible.”
CT103A is being evaluated as part of the multicenter phase 1/phase 2 FUMANBA-1 study in China for patients with relapsed or refractory multiple myeloma who received at least three prior lines of therapy.
Study data presented at last year’s ASH Annual Meeting and Exposition — based on median follow-up of 147 days — showed a 94.4% overall response rate and 50.7% complete response rate among 71 patients (median age 58 years; range, 41-71; 59.2% male) who received the investigational cell therapy.
Most patients (92.8%) achieved minimal residual disease-negative status by the first 1-month follow-up evaluation.
Most study participants (93%) developed cytokine release syndrome; however, only 2.8% of patients had grade 3 CRS. One patient (1.4%) developed grade 2 immune effector cell-associated neurotoxicity syndrome that resolved without treatment.
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:
Chunrui L, et al. Abstract 547. Presented at: ASH Annual Meeting and Exposition; Dec. 11-14, 2021; Atlanta.
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