The US Food and Drug Administration (FDA) has approved Orca-T (Tregzi), the first regulatory T cell-based immunotherapy for improving chronic graft-versus-host disease (GVHD)-free survival in adult patients with blood cancers undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT).
GVHD is a rare complication that can occur after a stem cell or bone marrow transplant. It is caused by the newly transplanted donor cells attacking the transplant recipient’s body. Symptoms may include skin rash, mouth sores, dry eyes, liver inflammation, development of scar tissue in the skin and joints, and damage to the lungs. The exact cause is unknown but likely results from a complex immune-mediated interaction between the donor and recipient cells.
Tregzi is a donor-derived cellular immunotherapy composed of three cell components: purified hematopoietic stem and progenitor cells (HSPCs), Treg cells, and conventional T cells, each derived from the blood of a closely matched donor. Tregzi is designed to reduce the risk of chronic GVHD.
The safety and efficacy of Tregzi was established through the PRECISION-T clinical trial (NCT05316701) in which 187 adult patients with blood cancers were randomly assigned to receive either Tregzi or a standard stem cell transplant. The primary endpoint was chronic GVHD-free survival, defined as the time from HSCT to the earliest occurrence of either death from any cause or the first onset of moderate or severe chronic GVHD, within two years after day 0.
At one year, 78% of patients who received Tregzi achieved the primary outcome, compared to 38.4% of patients who received a standard transplant. After accounting for death as a competing risk, 12.6% of patients who received Tregzi developed serious chronic GVHD within one year, compared with 44% of patients who received a standard transplant.
The adverse events observed with Tregzi were generally consistent with those expected in patients undergoing stem cell transplantation. The most commonly adverse event was infections. No patient had a severe reaction during the infusion of Tregzi, and no cases of graft failure were observed within the study period.
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To learn more about GVHD and rare cancers, visit https://checkrare.com/diseases/cancers/