FDA Grants Expedited Approval to Omidubicel for Patients With Hematologic Malignancies

On April 17, 2023, the Food and Drug Administration (FDA) granted expedited approval to omidubicel-onlv for 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. 

The FDA granted this application priority review, breakthrough designation and orphan drug designation.
 
The safety and efficacy of the treatment were evaluated in Study P0501, an open-label, multicenter, randomized trial of omidubicel transplantation or unmanipulated cord blood (UCB) unit transplantation following myeloablative conditioning in patients with hematologic malignancies. 
 
In total, 125 patients were randomized in this study, with 62 patients to the omidubicel group and 63 to the UCB group. There were 52 patients transplanted with omidubicel, receiving a median CD34+ cell dose of 9.0 X 106 cells/kg. In the UCB group, 56 patients were transplanted with 1 or 2 cord units (66% received 2 cord units). In the 42 patients with reported post-thaw cell dose, the median CD34+ cell dose was 0.2 X 106 cells/kg (range 0.0 to 0.8 X 106 cells/kg). Multiple conditioning regimens were used, including total body irradiation-based or chemotherapy-based options.
 
The main efficacy outcome measures of the study were time to neutrophil recovery following transplantation, as well as the incidence of Blood and Marrow Transplant Clinical Trials Network (BMT CTN) 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 (95% confidence interval [CI], 10 to 15 days) and 22 days in the UCB arm (95% CI, 19 to 25 days). In the omidubicel arrm, 87% of patients achieved neutrophil recovery, vs 83% percent in the UCB arm. The incidence of BMT CTN grade 2/3 bacterial or grade 3 fungal infections through Day 100 post transplantation was 39% and 60%, respectively, in the 2 groups.
 
Among 117 patients who received omidubicel for any disease, infusion reactions occurred in 47% of patients, acute GVHD in 58%, chronic GVHD in 35%, and graft failure in 3%. In patients with hematologic malignancies, the most common grade 3 to 5 adverse reactions were pain (33%), mucosal inflammation (31%), hypertension (25%), and gastrointestinal toxicity (19%). 
 
The prescribing information includes a Black Box label for fatal or life-threatening infusion reactions, graft-versus-host-disease, engraftment syndrome and graft failure.

Source:

FDA approves omidubicel to reduce time to neutrophil recovery and infection in patients with hematologic malignancies. Press Release. The US Food and Drug Administration. Published online April 17, 2023. Accessed April 18, 2023. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-omidubicel-reduce-time-neutrophil-recovery-and-infection-patients-hematologic