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W-02: Regulatory Implications for the Safety of CAR-T Gene Therapy

Poster Presenter

      Jaspal (JP) Ahluwalia

      • Medical Officer, OBE, CBER
      • FDA
        United States


The objective of this poster is the review the safety information available for CAR-T gene therapy and discuss why it required risk mitigation strategies beyond labeling to ensure the benefits outweigh the risks. for approval in the US.


We reviewed the clinical trial safety data for the pivotal trials for tisagenlecleucel and axicabtagene ciloleucel, the first two CAR-T gene therapies approved in the US. We discuss the unique risks which necessitated risk mitigation beyond product labeling that included boxed warning.


Clinical trial results from both tisagenlecleucel and axicabtagene ciloleucel showed a high percentage of patients developing cytokine release syndrome and/or neurological toxicities after being infused with the CAR-T products. The specific risk profile for these products required risk evaluation and mitigation strategy (REMS) with elements to assure safe use. The medical literature and experts in the field also identified the theoretical risk of secondary malignancy post-infusion for this class of products. Additionally, a postmarketing observational study was required further characterize this risk.


CAR-T gene therapy has a unique and severe risk profile which required a REMS with elements to assure safe use for approval.