FDA Approves First Regulatory T-Cell Therapy for Blood Cancer Patients Undergoing HSCT

The Food and Drug Administration approved Orca-T (Tregzi, Orca Bio), the first regulatory T (Treg) cell-based immunotherapy, to improve chronic graft-versus-host disease (GVHD)-free survival in adults with certain high-risk blood cancers undergoing allogeneic hematopoietic stem cell transplantation (HSCT).

Orca-T is a donor-derived cellular immunotherapy composed of three cell components: purified hematopoietic stem and progenitor cells, Treg cells and conventional T cells collected from an 8/8 HLA-matched donor. The therapy is designed to reduce the risk of chronic GVHD while supporting reconstitution of the patient's blood-forming and immune systems.  

FDA based its approval on findings from the randomized, multicenter PRECISION-T clinical trial, in which 187 adults with blood cancers were randomly assigned to receive Orca-T or standard stem cell transplant. At one year, 78% of patients who received Orca-T achieved GVHD-free survival compared with 38.4% of patients who received a standard transplant. After accounting for death as a competing risk, serious chronic GVHD occurred in 12.6% of patients who received Orca-T and 44% of patients in the standard transplant group within one year.

The application was granted Orphan Drug and Regenerative Medicine Advanced Therapy designations.