Omisirge: First FDA Approved Ex Vivo Expanded Cord Blood Graft for Stem Cell Transplantation

by Dr Debra Smith

On April 17, 2023, the FDA granted regular approval of Omisirge to Gamida Cell Ltd. Omisirge (omidubicel-onlv) is intended for use in patients 12 years and older with hematologic malignancies scheduled for umbilical cord transplantation following myeloablative conditioning. Each dose of Omisirge is patient-specific and contains cells from a healthy, allogeneic, pre-screened cord blood donor. The cell therapy product consists of two fractions derived from a single umbilical cord blood unit: a nicotinamide-modified cultured cell fraction containing hematopoietic stem and progenitor cells (HSPCs) and a non-cultured T-cell containing fraction. This therapy is intended to reduce the time to neutrophil recovery and reduce risk of infections after transplant.

FDA approval was based on Phase 3 clinical trial data (ClinicalTrials.gov Identifier: NCT02730299). The clinical trial included 125 patients randomized to receive omidubicel-only (n=62) or standard cord blood transplant (CBT; n=63). Median time to neutrophil engraftment was 12 days for the omidubicel-only arm compared to 22 days for the CBT arm. Recipients of the omidubicel-only graft also showed faster platelet recovery, lower incidence of early grade 2/3 bacterial or grade 3 invasive fungal infections through day 100, more time out of the hospital in the first 100 days post-transplant, and similar rates of GVHD and survival.

Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research (CBER), commented on the importance Omisirge and its approval.

"Today's approval is an important advance in cell therapy treatment in patients with blood cancer,” he said. “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."

References

  1. Horwitz ME, Stiff, PJ, Cutler C, et al. Omidubicel vs standard myeloablative umbilical cord blood transplantation: results of a phase 3 randomized study. Blood. 2021; 138(16):1429-1440.

  2. 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 May 22, 2023.