FDA Approves Omisirge, an Allogeneic Cell Therapy for Patients With Blood Cancers

ASCT remains the only curative therapy for patients with MDS.
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Omisirge is a nicotinamide modified allogeneic hematopoietic progenitor cell therapy derived from cord blood used as an allogeneic stem cell donor source.

The Food and Drug Administration (FDA) has approved Omisirge® (omidubicel-onlv) for use in adults and pediatric patients 12 years and older 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.

Omidubicel-onlv is a nicotinamide modified allogeneic hematopoietic progenitor cell therapy derived from cord blood. Treatment is administered as a single intravenous dose; each dose is patient-specific and contains healthy stem cells from an allogeneic pre-screened donor.

The approval was based on data from a multicenter, randomized, phase 3 trial (ClinicalTrials.gov Identifier: NCT02730299) that included patients 12 to 65 years of age with hematologic malignancies (eg, acute lymphoblastic leukemia, acute myeloid leukemia, myelodysplastic syndrome, chronic myeloid leukemia or lymphoma) who were eligible for allogeneic transplantation.

Patients were randomly assigned to receive omidubicel-onlv (n=62) or standard umbilical cord blood transplantation (UCBT; n=63). Efficacy was based on time to neutrophil recovery following transplantation and the incidence of Blood and Marrow Transplant Clinical Trials Network grade 2/3 bacterial or grade 3 fungal infections through day 100 following transplantation.

Results showed 87% of patients who received omidubicel-onlv achieved neutrophil recovery compared with 83% of those who received UCBT. The median time to neutrophil recovery was 12 days in the omidubicel-onlv arm and 22 days in the UCBT arm (P <.001). Grade 2/3 bacterial or grade 3 fungal infections through day 100 following transplantation occurred in 39% of patients in the omidubicel-onlv arm and 60% of patients in the UCBT arm.

The most common adverse reactions reported with omidubicel-onlv were infections, graft vs host disease (GvHD), and infusion reactions. The Omisirge labeling includes a Boxed Warning regarding the risk of infusion reactions, GvHD, engraftment syndrome, and graft failure. Due to the potential for severe adverse reactions, health care professionals should assess the risks and benefits of using the treatment.

“Today’s approval is an important advance in cell therapy treatment in patients with blood cancers,” said Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research. “Hastening the return of the body’s white blood cells can reduce the possibility of serious or overwhelming infection associated with stem cell transplantation. This approval reflects the FDA’s continued commitment to supporting development of innovative therapies for life-threatening cancers.”


1. FDA approves cell therapy for patients with blood cancers to reduce risk of infection following stem cell transplantation. News release. US Food and Drug Administration. Accessed April 17, 2023. https://www.fda.gov/news-events/press-announcements/fda-approves-cell-therapy-patients-blood-cancers-reduce-risk-infection-following-stem-cell
2. Omisirge. Package insert. Gamida Cell; 2023. Accessed April 17, 2023. http://www.gamida-cell.com/wp-content/uploads/Omisirge-final-PI.pdf