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, 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