The US Food and Drug Administration (FDA) has accepted for Priority Review Orca Bio’s Biologics License Application (BLA) for Orca-T, an investigational allogeneic T-cell immunotherapy being developed for the treatment of haematological malignancies including acute myeloid leukaemia (AML), acute lymphoblastic leukaemia (ALL) and myelodysplastic syndromes (MDS).

The company said the FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of 6 April 2026. If approved, Orca-T would be the first allogeneic T-cell immunotherapy available for the treatment of these blood cancers.

Nate Fernhoff, co-founder and chief executive officer at Orca Bio, said: “A stem cell transplant has been the only potentially curative option for many people with AML, ALL or MDS, however treatment-related toxicities too often hinder patient recovery. Acceptance of the Orca-T BLA marks a pivotal moment in our ability to deliver a first-in-class therapy designed to improve survival free from complications like graft versus host disease.”

Fernhoff added: “Supported by positive Phase 3 clinical data, today’s regulatory milestone reflects important recognition of the transformative potential of Orca-T. We look forward to working collaboratively with the FDA on the review of our application with the goal of advancing Orca-T and making it available to patients in need.”

The BLA submission is supported by results from the pivotal Phase 3 Precision-T (NCT04013685) study, a randomised, open-label, multi-centre trial comparing Orca-T with conventional allogeneic haematopoietic stem cell transplant (alloHSCT) in patients with AML, ALL and MDS. The study met its primary endpoint, demonstrating a statistically significant improvement in survival free of moderate-to-severe chronic graft versus host disease (cGvHD) in patients receiving Orca-T compared to alloHSCT.

Orca-T is composed of highly purified regulatory T-cells, haematopoietic stem cells and conventional T-cells derived from peripheral blood from matched related or unrelated donors. The therapy is designed to replace a patient’s diseased blood and immune system with a healthy one while reducing the risk of post-transplant complications. Orca-T has previously received both Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug Designation from the FDA for the prevention of graft versus host disease or death in transplant-eligible patients.

Based in Menlo Park, California, Orca Bio is a late-stage biotechnology company developing high-precision cell therapies for blood cancers and autoimmune diseases. Its proprietary manufacturing platform uses single-cell precision to generate cell products with defined composition and function. The company’s mission is to transform the field of curative allogeneic cell therapy and improve outcomes for patients with life-threatening diseases.