FDA approves Orca Bio cell therapy for certain blood cancers

The FDA approved Orca Bio’s Tregzi on June 30, clearing a new treatment for adults with certain blood cancers and making it the first regulatory T-cell-based immunotherapy to improve chronic graft-versus-host-disease-free survival after allogeneic stem-cell transplant. The decision gives patients with acute myeloid leukemia, acute lymphoblastic leukemia and myelodysplastic syndromes a new option.

Tregzi, also known clinically as Orca-T, is built from three cell components taken from an 8/8 HLA-matched related or unrelated donor: purified hematopoietic stem and progenitor cells, regulatory T cells and conventional T cells. The therapy is given after chemotherapy that prepares the body for a bone marrow or stem-cell transplant, with the aim of rebuilding a patient’s blood and immune system while reducing the graft-versus-host disease that can follow conventional donor transplants. That complication can damage organs, force long-term immune-suppressing treatment, raise infection risk and become fatal.

Orca Bio’s phase 3 Precision-T trial randomized 187 adults with blood cancers. The primary endpoint was chronic GVHD-free survival within two years after transplant. At one year, 78% of patients who received Tregzi were alive and free from moderate-to-severe chronic graft-versus-host disease, compared with 38% among those who received a conventional transplant. In patients over 50, that measure was 74% versus 35% with tacrolimus and methotrexate. Tregzi was also associated with lower rates of acute GVHD, non-relapse mortality, serious infections and rehospitalizations, along with better quality-of-life outcomes.

Tregzi will carry an acquisition cost of $428,000 for wholesalers, and Orca Bio expects orders by the end of July. The treatment is reserved for matched-donor transplant patients and requires a myeloablative preparative regimen.

Nate Fernhoff, Orca Bio’s chief executive: “This is going to be a really important option for leukemia patients.”