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FDA approves Orca-T, a novel allogeneic T-cell-based immunotherapy, for patients with hematological malignancies undergoing SCT

On June 30, 2026, the U.S. Food and Drug Administration (FDA) granted approval of Orca-T, an allogeneic T-cell-based immunotherapy, for the treatment of patients with hematological malignancies undergoing allogeneic stem cell transplantation (alloSCT).1

Although conventional alloSCT has provided a potentially curative option for patients with several hematologic malignancies, this therapeutic approach is associated with significant toxicities. Among these is graft-versus-host disease (GvHD), which can be life-threatening and may dramatically reduce a patient’s quality of life. For this reason, clinicians and patients must weigh up the benefits and curative potential of alloSCT with the associated risks.2

Orca-T is an allogeneic T-cell immunotherapy designed to reduce the risk of transplant-related complications, including GvHD. This high-precision immunotherapy is manufactured from related or unrelated matched donors and is composed of highly purified regulatory T-cells (Tregs), hematopoietic stem cells, and conventional T-cells.2 As opposed to traditional alloSCT infusion methods, with Orca-T, Tregs are infused two days prior to conventional T-cells.3 This allows time for Tregs to establish an immune barrier within patient tissues, thereby reducing the risk of GvHD.2

The approval of Orca-T is supported by data from the multicenter, randomized, Phase III Precision-T (NCT05316701) trial, evaluating the efficacy, tolerability, and safety of Orca-T compared with standard of care (SoC) alloSCT.3 187 adult patients with acute leukemia and myelodysplastic syndromes (MDS) were included in the trial and randomized 1:1 to receive Orca-T and tacrolimus (Orca-T plus TAC; n=93), or SoC allograft (n=94). The primary endpoint of the study was survival free from moderate-to-severe chronic GvHD, which was met in 78.0% (95% CI: 65–87) of patients receiving Orca-T compared with 38.4% (95% CI: 26–51) of patients receiving SoC (p<0.00001).

Additionally, several secondary endpoints were met, including improved GvHD and relapse-free survival (GRFS) rates of 63.1% for Orca-T (95% CI: 50–74) and 30.9% for SoC (95% CI: 20–42; p<0.001). Safety findings also remained consistent with previous studies, including a lower incidence of Grade 3 or higher infections in patients in the Orca-T arm compared with the SoC arm (8.4% and 16.1%, respectively).2,3

At SOHO 2025, we spoke with Rawan Faramand, MD, Moffitt Cancer Center, Tampa, FL, who shared insights into the clinical significance of outcomes from the Precision-T trial. Dr Faramand states, “with this strategy, we can improve immune reconstitution, therefore, lowering the rates of severe cGvHD, whilst still maintaining the graft-versus-leukemia effect.”

The approval of Orca-T provides a novel therapeutic option for patients with hematological malignancies, reducing the risks and burden of transplant-related complications, including GvHD. The results of the Precision-T trial also provide evidence for the efficacy of Treg-based immunotherapy, which warrants investigation in other hematological, genetic, and autoimmune diseases for which conventional alloSCT has been the primary curative treatment modality.3

References

  1. U.S. Food and Drug Administration. FDA approves new treatment that uses donor immune cells to prevent serious complications in blood cancer patients. Available here. (Last Accessed 01/07/2026).
  2. Orca Bio. Press Release: Orca Bio Announces Orca-T® Phase 3 Data Published in Blood Demonstrate Significant Improvement in Survival Free from Chronic Graft versus Host Disease in Patients with Hematological Malignancies. Available here. (Last accessed 01/07/2026).
  3. Meyer EH, Salhotra A, Gandhi AP, et al. Orca-T vs allogeneic hematopoietic stem cell transplantation (Precision-T): a multicenter, randomized phase 3 trial. Blood. 2026;147(11):1168-1177.

Written by Clare Harris

Edited by Anya Dragojlovic Kerkache & Natalie Markova