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Regenerative medicine advanced therapy and orphan drug designation given to ORCA-T

Oct 15, 2020

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The U.S. Food and Drug Administration (FDA) has granted both orphan drug designation and regenerative medicine advanced therapy (RMAT) to ORCA-T, an investigational engineered donor graft therapy.1 The RMAT designation allows for expediated development of new therapies and, as a cellular therapy, ORCA-T will receive early guidance from the FDA regarding manufacturing and clinical development. Orphan drug designation is granted to therapies that treat diseases affecting fewer than 200,000 people in the U.S.

ORCA-T is a T-cell depleted graft that is then reinfused with both purified regulatory T cells and conventional T cells. ORCA-T aims to allow for reconstitution of the immune system without triggering graft-versus-host disease.

The safety and efficacy of ORCA-T are currently being evaluated in a non-randomized, single-arm phase Ib trial (NCT04013685) in patients receiving myeloablative allogeneic hematopoietic stem cell transplantation for acute myeloid leukemia, acute lymphoid leukemia, mixed phenotype leukemia, or high-risk myelodysplastic syndromes.

More data on ORCA-T are expected to be announced at this year’s 62nd American Society of Hematology (ASH) annual meeting.

  1. Orca Bio. Orca Bio receives regenerative medicine advanced therapy (RMAT) designation for Orca-T. Published Oct 14, 2020. Accessed Oct 15, 2020.


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