FDA grants orphan drug designation to a multi-tumor-associated antigen (MultiTAA)-specific T-cell therapy for acute myeloid leukemia

On April 29, 2020, the United States Food and Drug Administration (FDA) granted orphan drug designation to a multi-tumour-associated antigen (MultiTAA)-specific T-cell product for the treatment of patients with acute myeloid leukemia (AML) after allogeneic stem cell transplant (allo-SCT).¹

MultiTAA is a novel, non-genetically modified, cell therapy approach that selectively expands patient tumor-specific T cells in vitro by presenting tumor-associated antigens and cytokines. While conventional CAR T-cell therapies recognize a single receptor, MultiTAA cell therapy is able to target a broad range of tumor antigens at the same time and does not require genetic modification. In addition, this is one of the first therapies to show epitope spreading (expansion of patients’ T cells recognizing tumor-specific antigens), which contributes to a lasting anti-tumor effect. The therapy is designed for administration in the outpatient setting.²

The results obtained in various liquid and solid tumors are promising; this MultiTAA-specific T-cell product was well tolerated and showed clinical benefit. A phase II study in patients with AML after allo-SCT will start soon.¹