The U.S. Food and Drug Administration has approved Tregzi, the first regulatory T (Treg) cell-based immunotherapy, to improve chronic graft-versus-host disease (cGVHD)-free survival in adult patients with blood cancers undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT).
“For patients with blood cancers who need stem cell transplantation, chronic graft-versus-host disease has long been one of the most feared and difficult-to-prevent complications,” said Karim Mikhail, B.Pharm., M.S., Acting Director of the FDA’s Center for Biologics Evaluation and Research (CBER). “Today’s approval offers a genuine new approach that can help reconstitute the immune system while substantially reducing that risk and reflects the promise of what cellular therapy can deliver for patients.”
Tregzi is a donor-derived cellular immunotherapy composed of three cell components: purified hematopoietic stem and progenitor cells, Treg cells, and conventional T cells, each collected from the mobilized peripheral blood of an 8/8 HLA-matched related or unrelated donor. Treg cells help regulate immune responses and maintain immune tolerance, and the product is designed to reduce the risk of chronic GVHD during reconstitution of the patient’s blood-forming and immune systems.
The approval is supported by PRECISION-T, a randomized trial in 187 adults with blood cancers including acute leukemia and myelodysplastic syndrome. Patients receiving Tregzi achieved significantly higher rates of chronic GVHD-free survival: 78% at one year compared with 38.4% in the standard transplant group. After accounting for death as a competing risk, 12.6% of Tregzi recipients developed serious chronic GVHD within one year, versus 44% of control patients.
The side effects observed with Tregzi were consistent with those expected in stem cell transplant recipients, most commonly infections. No patient had a severe reaction during infusion, and no cases of graft failure occurred within the study period. The FDA granted the application Orphan Drug and Regenerative Medicine Advanced Therapy designations.
The approval addresses an important unmet need in transplantation, where curing the cancer is often only part of the challenge; avoiding chronic GVHD is equally critical for long-term outcomes. Tregzi represents the first approved therapy in a new class of Treg cell-based immunotherapies.
Source: FDA Press Release
