FDA Approves Orca Bio's TREGZI as First Precision-Engineered Cell Therapy for Blood Cancer Transplants

The FDA has approved Orca Bio's TREGZI (Orca-T) as the first precision-engineered regulatory T-cell therapy for adult blood cancer patients undergoing stem cell transplants. In the Phase 3 Precision-T trial of 187 patients, TREGZI doubled the one-year chronic graft-versus-host disease-free survival rate to 78% compared to 38% for conventional transplants, while lowering non-relapse mortality to 3%. Orca Bio plans a controlled U.S. rollout at a wholesale acquisition cost of $428,000.

TREGZI FDA approval

• ▪The U.S. Food and Drug Administration approved Orca Bio's TREGZI, also known as Orca-T, on June 30, 2026
• ▪The U.S. Food and Drug Administration previously granted TREGZI both Orphan Drug Designation and Regenerative Medicine Advanced Therapy Designation
• ▪TREGZI is the first FDA-approved regulatory T cell-based immunotherapy for use in matched-donor hematopoietic stem cell transplantation in adults with hematological malignancies

Precision-T Phase 3 trial results

• ▪In the Precision-T trial, the chronic graft-versus-host disease-free survival rate at 12 months was 78% with TREGZI compared to 38% with conventional transplant
• ▪The Precision-T Phase 3 clinical trial evaluated 187 adult patients with acute myeloid leukemia, acute lymphoblastic leukemia, myelodysplastic syndrome, and mixed-phenotype acute leukemia

TREGZI cell composition

• ▪TREGZI is composed of hematopoietic stem and progenitor cells, highly purified regulatory T cells, and conventional T cells
• ▪TREGZI is a personalized treatment manufactured for each individual patient using living cells from a matched donor

Blood cancer transplant context

• ▪Conventional allogeneic stem cell transplants carry a high risk of chronic graft-versus-host disease, where donor immune cells attack the recipient's healthy tissues
• ▪Orca Bio set the wholesale acquisition cost of TREGZI at $428,000 and expects the therapy to be available for orders by late July 2026
• ▪Orca Bio plans a controlled U.S. commercial rollout of TREGZI, aiming to onboard 25 specialized transplant centers by the end of 2026