Omidubicel is a NAM-enabled stem cell therapy candidate under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers). Omidubicel is the first bone marrow transplant product to receive Breakthrough Therapy Designation from the U.S. Food and Drug Administration and has also received Orphan Drug Designation in the U.S. and EU.
Omidubicel has been studied in an international, multi-center, randomized Phase 3 clinical study evaluating its the safety and efficacy compared to standard umbilical cord blood transplant.
- The study achieved its primary endpoint (p<0.001). In the intent-to-treat analysis, median time to neutrophil engraftment was 12 days for patients receiving omidubicel (95% CI: 10-15 days) compared to 22 days for the comparator group (95% CI: 19-25 days).
- Omidubicel was generally well tolerated. Among patients who were transplanted per protocol, rates of acute and chronic graft-versus host disease were similar and cumulative incidence of infections was significantly smaller in omidubicel compared to controls for both viral infections and bacterial or invasive fungal infections.
- The study also met all three of its secondary endpoints, improving platelet engraftment, and reductions in infections and hospitalizations, which are key measures for success for bone marrow transplant.
The Phase 3 data also show that during the first 100 days of treatment, omidubicel-treated patients had significantly shorter durations of hospitalization, intensive care unit time, consultant visits, procedures, and transfusions than the control arm. These data provide further evidence of the clinical benefit associated with the more rapid hematopoietic recovery in patients treated with omidubicel and the corresponding reduction in healthcare resource utilization.
Full safety and efficacy data from this Phase 3 study have been published in Blood.
On August 1, the U.S. Food and Drug Administration accepted for filing the Company’s Biologics License Application for omidubicel for the treatment of patients with blood cancers in need of an allogenic hematopoietic stem cell transplant. The FDA granted Priority Review for the BLA and has set a target action date of January 30, 2023.