The US Food and Drug Administration (FDA) has approved Omisirge (omidubicel-onlv), a nicotinamide-modified allogeneic hematopoietic progenitor cell therapy derived from cord blood.
The therapy is intended to reduce the time to neutrophil recovery and risk of infection in patients with hematologic malignancies who are 12 years of age and older and are set to undergo cord blood transplant with myeloablative conditioning.
Omidubicel-onlv is administered as a single intravenous dose. Each dose is patient-specific and contains stem cells from a pre-screened cord blood donation.
The approval of this therapy was based on data from a 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 transplant.
Patients were randomly assigned to receive omidubicel-onlv (n=62) or standard cord blood transplant (CBT; n=63). Efficacy was based on time to neutrophil recovery and the incidence of grade 2/3 bacterial or grade 3 fungal infections through day 100 after transplant.
Results showed that 87% of patients who received omidubicel-onlv achieved neutrophil recovery, as did 83% of those who received CBT. The median time to neutrophil recovery was 12 days in the omidubicel-onlv arm and 22 days in the CBT arm (P <.001). Grade 2/3 bacterial or grade 3 fungal infections through day 100 occurred in 39% of patients in the omidubicel-onlv arm and 60% of patients in the CBT 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.
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
This article originally appeared on MPR