FDA D.I.S.C.O. Burst Edition: FDA approval of Omisirge (omidubicel-onlv) 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) for adults and pediatric patients aged ≥12 years with hematologic malignancies planned for umbilical cord blood transplantation after myeloablative conditioning, to reduce time to neutrophil recovery and incidence of infection. Approval was supported by Study P0501, which reported median neutrophil recovery of 12 vs 22 days (omidubicel-onlv vs unmanipulated cord blood) and Day-100 specified infection rates of 39% vs 60%.
FDA approval (April 17, 2023): Omisirge (omidubicel-onlv)
The FDA approved omidubicel-onlv (Omisirge) for adult and pediatric patients aged 12 years and older with hematologic malignancies who are planned for umbilical cord blood transplantation following myeloablative conditioning, to reduce time to neutrophil recovery and the incidence of infection.
Key evidence
Safety and efficacy were evaluated in Study P0501, an open-label, multicenter, randomized trial comparing omidubicel-onlv transplantation vs unmanipulated cord blood unit transplantation after myeloablative conditioning.
- Randomized: 125 patients (62 to omidubicel-onlv; 63 to unmanipulated cord blood)
- Transplanted: 52 received omidubicel-onlv; 56 received unmanipulated cord blood (one or two cord units)
- Conditioning regimens included total body irradiation–based or chemotherapy-based options.
Primary efficacy outcomes:
- Median time to neutrophil recovery: 12 days (omidubicel-onlv) vs 22 days (unmanipulated cord blood)
- Neutrophil recovery achieved: 87% vs 83%
- BMT CTN Grade 2/3 bacterial or Grade 3 fungal infections through Day 100: 39% vs 60%
Safety
Prescribing information includes a Boxed Warning for fatal or life-threatening infusion reactions, graft-versus-host disease, engraftment syndrome, and graft failure. Among 117 patients who received 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, mucosal inflammation, hypertension, and gastrointestinal toxicity.