Zedenoleucel (MT-401) granted orphan drug designation by the EMA

On July 10, 2023, zedenoleucel (MT-401), a multi-tumor-associated antigen (multiTAA)-specific T-cell product, which was previously covered by the AML Hub, was granted orphan drug designation by the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA), for the treatment of patients with acute myeloid leukemia (AML).¹ Zedenoleucel previously received orphan drug designation from the U.S. Food and Drug Administration (FDA) on April 29, 2020, for the treatment of patients with AML post-allogeneic hematopoietic stem cell transplantation.¹

Zedenoleucel is a novel, non-genetically modified cell therapy that selectively expands a patient’s T cells to target multiple antigens expressed on tumor cells.¹ Zedenoleucel is currently being investigated in a phase II clinical trial (NCT04511130) for the treatment of patients with relapsed AML who have undergone allogeneic hematopoietic stem cell transplantation.¹ Promising initial results have been observed in this trial in patients with measurable residual disease, indicating that zedenoleucel has potential as a treatment for patients with AML prior to relapse.¹