Omidubicel receives FDA approval for patients with hematological malignancies requiring stem cell transplantation

On April 17, 2023, the U.S. Food and Drug Administration (FDA) approved omidubicel, an allogeneic cord blood-derived cell therapy, for patients with hematological malignancies requiring transplantation. This product is intended for adult and pediatric patients aged 12 and over and aims to speed up the recovery of neutrophils and reduce the risk of infection.1

Allogeneic stem cell transplantation (alloSCT) represents a promising treatment strategy for patients with a variety of blood cancers, and umbilical cord blood (UCB) is a valuable source of stem cells.2 Despite the success of UCB transplantation, there are some limitations to this approach, including low stem cell yields and delayed hematopoietic recovery. 2, 3 Furthermore, patients undergoing alloSCT may have a weakened immune system and thus be at a higher risk of severe infections.1

Omidubicel is a novel, UCB-derived cellular therapy product consisting of an ex-vivo nicotinamide-expanded CD133+ fraction and a non-expanded CD133fraction.2 Culturing stem cells in the presence of nicotinamide has shown to inhibit stem cell differentiation, enhance the functionality of hematopoietic stem cells, and improve engraftment. 2, 4 The approval of omidubicel is based on a randomized Phase III study comparing transplantation with omidubicel to standard UCB transplantation in 125 patients (NCT02730299). Primary outcome measures included time to neutrophil engraftment, and results demonstrated that 87% of patients randomized to receive omidubicel achieved neutrophil recovery at a median of 12 days, versus 83% of patients who were randomized to receive UCB transplantation and achieved neutrophil recovery with a median of 22 days .1 Side effects commonly associated with the use of omidubicel include graft-versus-host disease (GvHD), engraftment syndrome, graft failure, and infusion reactions, which patients should be regularly monitored for.1

At the Tenth Annual Meeting of the Society of Hematologic Oncology (SOHO), Chenyu Lin, MD, Duke University Medical Center, Durham, NC, discussed the findings from a pooled analysis of five multi-center clinical trials evaluating the long-term outcomes of transplantation with omidubicel. Dr Lin states that, “[Omidubicel] does have the potential to become the first expanded cell therapy product to be approved for use in allogeneic stem cell transplantation, which would be a phenomenal success and a significant benefit to many of our patients who are lacking appropriate stem cell donors.”

Omidubicel is the first allogeneic cell therapy approved for alloSCT and represents a valuable novel approach for patients with blood cancers. 


  1. U.S. Food and Drug Administration. FDA Approves Cell Therapy for Patients with Blood Cancers to Reduce Risk of Infection Following Stem Cell Transplantation. Available from:,reduce%20the%20risk%20of%20infection. (Last accessed 13/04/2023) 
  2. Horwitz ME, Stiff PJ, Cutler C, et al. Omidubicel vs standard myeloablative umbilical cord blood transplantation: results of a phase 3 randomized study. Blood. 2021 Oct 21;138(16):1429-1440. 
  3. Cohen Y, Nagler A. Umbilical cord blood transplantation–how, when and for whom? Blood Reviews. 2004 Sep;18(3):167-79. 
  4. Lin C, Sajeev G, Stiff PJ, et al. Health-Related Quality of Life Following Allogeneic Hematopoietic Cell Transplantation with Omidubicel versus Umbilical Cord Blood. Transplantation and Cellular Therapy. 2023 Jan;29(1):52.e1-52.e9. 

Written by Anya Dragojlovic-Kerkache
Edited by Thomas Southgate