Omidubicel

Omidubicel is a nicotinamide-modified allogeneic hematopoietic progenitor cell therapy derived from cord blood used as an allogeneic stem cell donor source. Umbilical blood cord cells are expanded ex-vivo and nicotinamide (a form of vitamin B3) is used as an active agent in the culture system to inhibit the differentiation and increase the functionality of hematopoietic stem and progenitor cells. This type of cell therapy benefits patients that require stem cell transplantation as a treatment for blood cancers for whom a related or unrelated donor cannot be found due to the requirement of high-grade human leukocyte antigen (HLA)-matching. In this group of patients, umbilical cord blood can be used as an alternative cell source since there is a lower risk of graft failure or graft-versus-host disease, even with low-grade HLA-matching.

A pooled analysis of five prospective clinical trials found that the use of omidubicel led to stable trilineage hematopoiesis, immune competence, and graft durability in extended follow-up. In addition, the fact that 39% of the study's cohort were non-White patients highlights the importance of this type of treatment in key demographics with low donor availability. In April 2023, the FDA approved the use of omidubicel in adults and pediatric patients 12 years and older with blood cancers planned for umbilical cord blood transplantation following a myeloablative conditioning regimen. It is administered as a single intravenous dose specific to each patient.