Omidubicel-onlv has recently received US Food and Drug Administration (FDA) approval for infection prevention in the context of stem cell transplantation in patients with hematological malignancies such as diffuse large B-cell lymphoma (DLBCL) and myelofibrosis (MF), according to a recently published press release.

Stem cell transplantation in patients with hematological malignancies is often preceded by radiation or chemotherapy, leading to severe immunosuppression; therefore, infection prevention is of great interest in these patients.

The stem cell-based therapy increases the migration and engraftment of progenitor stem cells after a stem cell transplant through nicotinamide-mediated cell differentiation inhibition. Rapid neutrophil engraftment significantly reduces the risk of infection in otherwise immunocompromised patients.

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The effectiveness of the treatment has been recently tested in the phase 3 randomized, double-blind, multicenter clinical trial, ADVANCE 4, which included 125 patients with different hematological neoplasms such as DLBCL and MF who underwent stem cell transplantation. Omidubicel was administered as a single intravenous infusion.

The results showed that omidubicel administration led to neutrophil engraftment in 12 days, while patients in the control group experienced engraftment after 22 days. Furthermore, bacterial and fungal infections during the first 100 days after transplantation were close to 20% more common within the control group.

Patients who received omidubicel spent fewer days in the intensive care unit and required fewer blood transfusions than patients in the control group. Consultant visits were also less frequent in the omidubicel group.

The most common adverse effects associated with treatment included engraftment syndrome, graft failure, and infusion reactions.

“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,” Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, said.


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 (FDA); April 17, 2023.