FDA approves omidubicel to reduce time to neutrophil recovery and infection in patients with hematologic malignancies

Regulatory approval: On April 17, 2023, the FDA approved omidubicel-onlv (Omisirge; Gamida Cell Ltd.) for adults and pediatric patients ≥12 years with hematologic malignancies planned for umbilical cord blood transplantation after myeloablative conditioning. The approval is to reduce time to neutrophil recovery and the incidence of infection. The decision was supported by the open-label, multicenter, randomized Study P0501 (NCT02730299) comparing omidubicel-onlv transplantation with unmanipulated cord blood transplantation.

FDA approval (April 17, 2023)

The FDA approved omidubicel-onlv (Omisirge; Gamida Cell Ltd.) for adult and pediatric patients (≥12 years) with hematologic malignancies who are planned for umbilical cord blood transplantation following myeloablative conditioning, to reduce time to neutrophil recovery and incidence of infection.

Supporting evidence: Study P0501 (NCT02730299)

Study P0501 was an open-label, multicenter, randomized trial comparing omidubicel-onlv transplantation vs unmanipulated cord blood (UCB) transplantation after myeloablative conditioning.

  • Randomized: 125 patients (62 omidubicel-onlv; 63 UCB)
  • Transplanted: 52 received omidubicel-onlv; 56 received UCB (66% received two cord units)

Efficacy endpoints included:

  • Time to neutrophil recovery: median 12 days (95% CI 10–15) with omidubicel-onlv vs 22 days (95% CI 19–25) with UCB; neutrophil recovery achieved in 87% vs 83%, respectively.
  • Infections through Day 100 (BMT CTN grade 2/3 bacterial or grade 3 fungal): 39% with omidubicel-onlv vs 60% with UCB.

Safety

Prescribing information includes a Boxed Warning for fatal or life-threatening infusion reactions, GvHD, engraftment syndrome, and graft failure. Among 117 patients receiving omidubicel-onlv for any disease: infusion reactions 47%, acute GVHD 58%, chronic GVHD 35%, graft failure 3%. In Study P0501, common grade 3–5 adverse reactions included pain (33%), mucosal inflammation (31%), hypertension (25%), and gastrointestinal toxicity (19%).

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