Dr Horwitz on the FDA Approval of Omidubicel in Blood Cancers Requiring Transplant

Mitchell E. Horwitz, MD, professor, medicine, hematologic malignances, and cellular therapy, member, Duke Cancer Institute, affiliate, Duke Regeneration Center, discusses the significance of omidubicel-onlv (Omisirge) as an FDA-approved treatment option to reduce the risk of infection in patients with blood cancers and highlights key findings from the pivotal phase 3 trial (NCT02730299).

Dr Horwitz on Omidubicel vs SOC in Blood Cancers

On April 17, 2023, the FDA approved omidubicel, a donor cord blood–based cell therapy, in adult and pediatric patients at least 12 years of age with blood cancers who have received a myeloablative conditioning regimen and are planned to receive umbilical cord blood transplantation. This approval was backed by findings from the phase 3 trial, in which 125 patients with blood cancers were randomized to receive omidubicel or standard-of-care (SOC) umbilical cord blood transplantation. In total, 87% of the patients who received omidubicel achieved neutrophil recovery at a median of 12 days post–omidubicel infusion vs 83% of patients who received SOC transplantation and achieved neutrophil recovery at a median of 22 days post-transplant.

Omidubicel is the first modified allogeneic cord blood–based stem cell graft to gain FDA approval, Horwitz says. This regulatory decision is unprecedented and provides a new manipulated stem cell treatment option for patients with blood cancers requiring umbilical cord blood transplantation, Horwitz explains.

Dr Horwitz on the Benefits of Omidubicel in Blood Cancers

Because omidubicel induces rapid neutrophil engraftment, patients in the phase 3 study who received omidubicel spent significantly less time in the hospital compared with those who received SOC transplant, Horwitz emphasizes. In this multicenter study, during the first 100 days after treatment, the patients who received omidubicel spent a median of 48 days in the hospital vs 61 days for those who received SOC transplantation.

The benefits of omidubicel extend beyond neutrophil engraftment, as the patients who received this agent also experienced significantly faster platelet recovery and fewer bacterial and fungal infections than those who received SOC, Horwitz notes. Unexpectedly, the patients in the omidubicel arm also had fewer viral infections than those in the SOC arm, which studies of immune reconstitution will evaluate further, Horwitz concludes.