FDA Brief: Cell Therapy Approved to Reduce Infection Risk After Stem Cell Transplantation

By: JNCCN 360 Staff
Posted: Tuesday, April 18, 2023

On April 17, the U.S. Food and Drug Administration (FDA) approved omidubicel-onlv (Omisirge), a substantially modified allogeneic (donor) cord blood–based cell therapy, to quicken the recovery of neutrophils and reduce the risk of infection. The product is intended for use in adults and pediatric patients 12 years and older with hematologic malignancies planned for umbilical cord blood transplantation after a myeloablative conditioning regimen.

Administered as a single intravenous dose, omidubicel is composed of human allogeneic stem cells from umbilical cord blood that are processed and cultured with nicotinamide. Each dose is patient-specific, containing healthy stem cells from an allogeneic prescreened donor.

The safety and effectiveness of omidubicel were supported by a randomized, multicenter study comparing transplantation of omidubicel with transplantation of umbilical cord blood, in 125 patients between the ages of 12 and 65. All study patients had confirmed hematologic malignancies. The efficacy of omidubicel was based on the amount of time needed for recovery of the subject’s neutrophils and the incidence of infections after transplantation.

A total of 87% of patients randomly assigned to receive omidubicel achieved neutrophil recovery, with a median of 12 days after treatment with the product, compared with 83% of those who were randomly assigned to receive umbilical cord blood transplantation and achieved neutrophil recovery, with a median of 22 days. Bacterial or fungal infections by 100 days following transplantation were seen in 39% of those receiving omidubicel versus 60% of those in the control group who received umbilical cord blood.

Like all approved umbilical cord products, the label for omidubicel carries a boxed warning for infusion reactions, graft-versus-host disease, engraftment syndrome, and graft failure. The most common adverse reactions associated with omidubicel included infections, graft-versus-host disease, and infusion reactions. Patients who receive omidubicel should be monitored for signs and symptoms of infusion reactions, graft-versus-host disease, engraftment syndrome, graft failure, transmission of serious infections or rare genetic diseases from the donor cells, as well as lifelong for secondary malignancies.

U.S. Food and Drug Administration

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