FDA Approves Tregzi Cell Therapy to Prevent Chronic GVHD in Blood Cancer Patients
The FDA has approved Tregzi, a donor-derived T-cell immunotherapy, to reduce the risk of chronic graft-versus-host disease following stem cell transplant in adults with high-risk blood cancers. In a pivotal trial, 78% of recipients were alive and free of severe chronic GVHD at one year versus 38.4% with standard transplant.
The U.S. Food and Drug Administration (FDA) has approved Tregzi (allogeneic regulatory T-cell immunotherapy, formerly Orca-T) for adults with higher-risk blood cancers such as acute lymphoblastic leukemia (ALL). The therapy is intended to improve survival free of chronic graft-versus-host disease (GVHD), a severe complication of stem cell transplantation.
The product is assembled from three donor-derived cell populations: hematopoietic stem and progenitor cells (HSPCs), regulatory T cells (Tregs), and conventional T cells, all from a single 8/8 HLA-matched donor. Tregs help balance immune responses and reduce the risk of chronic GVHD, while HSPCs enable the patient to rebuild blood and immune function.
Approval was supported by the randomized PRECISION-T study in 187 adults with blood cancers including ALL and myelodysplastic syndromes (MDS). At one year post-transplant, 78% of patients receiving Tregzi remained alive and free of moderate or severe chronic GVHD, compared to 38.4% of those who received a conventional stem cell transplant. Accounting for death as a competing risk, the incidence of serious chronic GVHD was 12.6% in the Tregzi arm versus 44% in the control arm.
The FDA’s acting director of the Center for Biologics Evaluation and Research noted that chronic GVHD “has long been one of the most feared and difficult-to-prevent complications” following transplant.
Adverse events were largely consistent with typical post-transplant complications, primarily infections. No severe infusion reactions or graft failures were observed.
Tregzi holds both Orphan Drug and Regenerative Medicine Advanced Therapy designations from the FDA.