Omidubicel-onlv granted FDA approval for patients with hematologic malignancies eligible for umbilical cord blood transplantation

On April 17, 2023, omidubicel-onlv, a substantially modified allogeneic cord blood-based cell therapy, which has been discussed previously on the AML Hub here, here and here, was approved by the U.S. Food and Drug Administration (FDA) for the treatment of adult and pediatric patients aged ≥12 years with hematologic malignancies who are eligible for umbilical cord blood transplantation following myeloablative conditioning.¹ Omidubicel-onlv contains stem cells derived from umbilical cord blood from an allogeneic pre-screened donor that are processed and cultured with nicotinamide before being administered as a single intravenous dose.¹

This approval is based on results from a phase III randomized, multicenter study (NCT02730299) demonstrating efficacy and an acceptable safety profile of omidubicel-onlv compared with standard umbilical cord blood transplantation in 125 patients aged 12–65 years with acute lymphoblastic leukemia, acute myeloid leukemia, chronic myeloid leukemia, myelodysplastic syndromes, or lymphoma.¹ The trial notably included a high proportion of patients who were non-white (44%), a historically underrepresented population in registries of unrelated adult donors and umbilical cord blood.²

• Neutrophil recovery was achieved by 87% of patients who received omidubicel-onlv versus 83% of patients who received standard umbilical cord blood transplantation.
  • Median time to neutrophil recovery was 12 days for patients who received omidubicel-onlv versus 22 days for those who received standard umbilical cord blood transplantation.
• Patients who received omidubicel-onlv were found to have lower incidences of bacterial and fungal infections at 100 days post-transplantation (39%) compared with patients who received standard umbilical cord blood transplantation (60%).
• The most commonly reported adverse events were infections, graft-versus-host disease, and infusion reactions.

As with all umbilical cord products, omidubicel-onlv has a Boxed Warning for infusion reactions, graft-versus-host disease, engraftment syndrome, and graft failure.¹