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
Welcome back to the D.I.S.C.O., FDA’s Drug Information Soundcast in Clinical Oncology, Burst Edition, brought to you by FDA’s Division of Drug Information in partnership with FDA’s Oncology Center of Excellence. Today we’ll provide a quick update on a recent FDA cancer therapeutic approval.
On April 17, 2023, the FDA approved omidubicel-onlv, brand name Omisirge, for use in adult and pediatric patients 12 years and older with hematologic malignancies who are planned for umbilical cord blood transplantation following myeloablative conditioning to reduce the time to neutrophil recovery and the incidence of infection.
Safety and efficacy were evaluated in Study P0501, an open-label, multicenter, randomized trial of omidubicel-onlv transplantation or unmanipulated cord blood unit transplantation following myeloablative conditioning in patients with hematologic malignancies. In total, 125 patients were randomized, 62 patients to receive omidubicel-onlv and 63 to the unmanipulated cord blood group. Fifty-two patients were transplanted with omidubicel-onlv. Fifty-six patients were transplanted in the unmanipulated cord blood arm with one or two cord units. Multiple conditioning regimens were used, including Total Body Irradiation-based or chemotherapy-based options.
The main efficacy outcome measures were time to neutrophil recovery following transplantation and the incidence of Blood and Marrow Transplant Clinical Trials Network Grade 2/3 bacterial or Grade 3 fungal infections through Day 100 post transplantation. The median time to neutrophil recovery was 12 days for those receiving omidubicel-onlv and 22 days in the unmanipulated cord blood arm. Eighty-seven percent in the omidubicel-onlv arm and 83% in the unmanipulated cord blood arm achieved neutrophil recovery. The incidence of Blood and Marrow Transplant Clinical Trials Network Grade 2/3 bacterial or Grade 3 fungal infections through Day 100 post transplantation was 39% and 60%, respectively, in the two groups.
Similar to approved unmanipulated cord blood products, the prescribing information contains 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 occurred in 47% of patients, acute graft versus host disease in 58%, chronic graft versus host disease in 35%, and graft failure in 3%.
In patients with hematologic malignancies in Study P0501, the most common Grade 3-5 adverse reactions were pain, mucosal inflammation, hypertension, and gastrointestinal toxicity.
Full prescribing information for these approvals can be found on the web at www.fda.gov/, key word search Approved Cellular and Gene Therapy Products.
Health care professionals should report serious adverse events to FDA’s MedWatch Reporting Program at www.fda.gov/medwatch.
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