On April 17, 2023, omidubicel-onlv was approved for use in adult and pediatric patients (≥ 12 years) with hematologic malignancies who are planned for umbilical cord blood transplantation following myeloablative conditioning to reduce the time to neutrophil recovery and the incidence of infection.1 Omidubicel is a nicotinamide-modified allogeneic hematopoietic progenitor cell therapy derived from cord blood used as an allogeneic stem cell donor source.
Supporting Efficacy Data
Approval was based on findings in the multicenter open-label Study P0501 (ClinicalTrials.gov identifier NCT02730299), in which 125 patients were randomized to omidubicel transplantation (n = 62) or unmanipulated cord blood unit transplantation (n = 63). A total of 52 and 56 patients underwent transplantation; 5 patients in the omidubicel group did not receive the product because of manufacturing failure.
Omidubicel-omlv has boxed warnings for infusion reactions, graft-vs-host disease, engraftment syndrome, and graft failure. It also has warnings/precautions for malignancies of donor origin, transmission of serious infections, and transmission of rare genetic diseases.
On intent-to-treat analysis, the median time to neutrophil recovery was 12 days (95% confidence interval [CI] = 10–15 days) in the omidubicel group and 22 days (95% CI = 19–25 days) in the unmanipulated cord blood–group (absolute difference = 10 days [95% CI = 6–14 days]). Overall, neutrophil recovery was achieved in 87% vs 83% of patients. The incidence of Blood and Marrow Transplant Clinical Trials Network grade 2 or 3 bacterial or grade 3 fungal infections through day 100 posttransplantation was 39% vs 60% (absolute difference = 22%, 95% CI = 4%–39%).
How It Is Used
The recommended omidubicel dose is two sequential infusions consisting of the following: a cultured fraction consisting of a minimum of 8.0 × 108 total viable cells with a minimum of 8.7% CD34-positive cells and a minimum of 9.2 × 107 total CD34-positive cells; followed by a noncultured fraction consisting of a minimum of 4.0 × 108 total viable cells with a minimum of 2.4 × 107 CD3-positive cells.
Among all patients who underwent transplantation in Study P0501, the most common grade ≥ 3 adverse events in the omidubicel group were pain, mucosal inflammation, hypertension, and gastrointestinal toxicity. Grade II to IV acute graft-vs-host disease occurred in 62% vs 43%. Fatal adverse events occurred in 17% of patients in the omidubicel group and in 29% of patients of the unmanipulated cord blood–group.
Omidubicel has boxed warnings for infusion reactions, graft-vs-host disease, engraftment syndrome, and graft failure. It also has warnings/precautions for malignancies of donor origin, transmission of serious infections, and transmission of rare genetic diseases.
1. Omisirge (omidubicel-onlv) suspension for infusion, for intravenous use, prescribing information, Gamida Cell, Inc, April 2023. Available at https://www.fda.gov/media/167202/download. Accessed April 25, 2023.