The US Food and Drug Administration has approved Tregzi, the first regulatory T-cell-based immunotherapy indicated to improve chronic graft-versus-host disease-free survival in adult patients with blood cancers undergoing allogeneic hematopoietic stem cell transplantation, according to a US Food and Drug Administration press release.
Tregzi is a donor-derived cellular immunotherapy composed of purified hematopoietic stem and progenitor cells, regulatory T cells, and conventional T cells collected from the mobilized peripheral blood of an 8/8 human leukocyte antigen-matched related or unrelated donor. According to the US Food and Drug Administration (FDA), patients receive the therapy after chemotherapy to prepare for a bone marrow or stem cell transplant. The agency states that regulatory T cells help regulate immune responses and maintain immune tolerance and that Tregzi is designed to reduce the risk of chronic graft-versus-host disease (GVHD) during reconstitution of the blood-forming and immune systems.
The approval was based on the PRECISION-T trial, which enrolled 187 adult patients with blood cancers, including acute leukemia and myelodysplastic syndrome. Patients were randomly assigned to receive either Tregzi or a standard stem cell transplant. The primary endpoint was chronic GVHD-free survival, measured from transplantation until death from any cause or the first occurrence of moderate or severe chronic GVHD within 2 years.
One-year chronic GVHD-free survival was 78% with Tregzi compared with 38% with a standard transplant. Accounting for death as a competing risk, serious chronic GVHD occurred within 1 year in 13% of patients treated with Tregzi compared with 44% of those who received a standard transplant.
According to the FDA, the most common side effects were infections. No severe infusion reactions or cases of graft failure were reported during the study period. Tregzi previously received Orphan Drug and Regenerative Medicine Advanced Therapy designations. The FDA granted approval to Orca Biosystems, Inc.
Source: US Food and Drug Administration
