FDA’s authorisation based on data showing the precision-engineered cell therapy improved survival free of GVHD in adults with haematological malignancies.
The US Food and Drug Administration (FDA) has approved the first precision-engineered cell therapy for allogeneic transplant in adults with haematological malignancies.
This authorisation of Orca Bio’s Tregzi (allogeneic regulatory T cell immunotherapy with HSPC and T cells-vldq), also known as Orca-T, represents the agency’s first approval of a treatment that uses highly purified Tregs.
The personalised cell therapy is designed for matched-donor haematopoietic stem cell transplantation with myeloablative preparative regimen, for hematopoietic and immunologic reconstitution and to improve chronic graft-versus-host disease (GVHD)-free survival (cGFS), in adults with haaematological malignancies.
FDA’s approval of Tregzi is based on results from the Precision-T Phase III study in acute myeloid leukaemia (AML), acute lymphoblastic leukaemia (ALL), myelodysplastic syndrome (MDS) and mixed-phenotype acute leukaemia (MPAL).
At 12 months, compared to a conventional allogeneic haematopoietic stem cell transplant (alloHSCT) plus TAC/methotrexate (TAC/MTX), Tregzi plus single-agent tacrolimus (TAC) delivered:
- Chronic cGFS at 78 percent with Tregzi compared to 38 percent with alloHSCT
- A rate of chronic GVHD of 13 percent and 44 percent with Tregzi and alloHSCT
- Overall survival of 94 percent with Tregzi and 83 percent with alloHSCT.
Furthermore, one of the key safety findings showed that the total incidence of Grade 3 or 4 acute GVHD at day +180 with Tregzi was six percent versus 10 percent with alloHSCT.
Dr Robert Negrin, Professor of Medicine, Blood And Marrow Transplantation at Stanford Medicine, said: “Developing this concept from early foundational research in our labs based upon the fundamental biology of regulatory T cells, to it now receiving the first FDA approval for a therapy that utilises highly purified Tregs, is a defining moment for the transplant community.
“The peer-reviewed findings demonstrated this precision-engineered cell therapy delivered improved GVHD-free survival alongside less toxicity, including fewer serious infections and lower non-relapse mortality.”
[The FDA’s approval of Tregzi] signals a new era in transplant medicine [with] this precision-engineered cell therapy… equipping providers with a new option to reduce serious toxicities and improve treatment outcomes”
Dr Miguel-Angel Perales, Memorial Sloan Kettering Cancer Center
Dr Miguel-Angel Perales, Medical Oncologist and Chief of the Adult Bone Marrow Transplant Service at Memorial Sloan Kettering Cancer Center, said: “For transplant physicians, one of our greatest challenges has long been preserving the vital graft-versus-leukaemia effect while minimising the risk of GVHD and infection.”
As such, the FDA’s decision for Tregzi “signals a new era in transplant medicine. This precision-engineered cell therapy is built on the foundational principles established by our early CD34 cell selection work and can now be delivered at scale, equipping providers with a new option to reduce serious toxicities and improve treatment outcomes.”
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