Omidubicel BLA Accepted With Priority Review in Hematological Malignancies


Omidubicel demonstrated neutrophil engraftment a median of 10 days faster than standard UCBT.

The FDA has accepted a biologics license application (BLA) with priority review for omidubicel, an allogeneic hematopoietic stem cell transplant candidate developed by Gamida Cell for the treatment of hematological cancers.1 The BLA was supported by results reported in Blood from the phase 3 clinical trial (NCT02730299), which indicated that patients treated with omidubicel demonstrated faster hematopoietic recovery and had fewer early transplant-related complications compared with patients in the control arm treated with standard umbilical cord blood transplantation (UCBT).2

Patients in the omidubicel arm of the study had a faster median time to neutrophil engraftment (12 days vs 22 days, 95% CI, P <.001), a 96% cumulative incidence of neutrophil engraftment (vs 89% for the control arm), faster platelet recovery (55% vs 35% recovery by 42 days; P = .028),and had a higher median number of days out of the hospital in the first 100 days after transplant (61 vs 48 days; P = .005). Additionally, a lower incidence of first grade 2 to 3 bacterial or invasive fungal infections was observed in the omidubicel arm (37%) compared to the control arm (57%) (P = .027).

“The FDA’s acceptance of our BLA with Priority Review signifies a critical milestone in our mission to deliver a new stem cell therapy option for patients in need of a donor for an allogeneic stem cell transplant,” Julian Adams, PhD, chief executive officer of Gamida Cell, said in a statement.1 “We are encouraged by the positive and sustained follow-up results from patients participating in the Phase 3 trial of omidubicel, including a positive overall survival trend one-year out from treatment. These results provide promising rationale that, if approved, omidubicel could become a treatment of choice for patients in need of an allo-HSCT transplant. We look forward to working with the FDA throughout the review process to bring omidubicel to patients as quickly as possible.”

The study enrolled 125 patients aged 13 to 65 years (median age, 41) with hematological malignancies. Of these patients, 48% had acute myeloid leukemia and 33% had acute lymphoblastic leukemia. Ten patients in the omidubicel arm did not receive a transplant per protocol, while 8 patients in the control arm also did not receive a transplant per protocol. The patients included in the study were racially diverse: 16% black, 14% Asian, 3% multiracial, and 13% Hispanic or Latino.

“The encouraging results of transplantation with omidubicel have important implications for minority populations in need of allo-HSCT,” first author Mitchell E. Horwitz, MD, Duke University Medical Center, and colleagues noted.2 “Forty-four percent of the patients treated on study were non-White, a population known to be underrepresented in the world-wide unrelated adult donor and UCB registries. The underrepresentation of UCB grafts from Black donors has necessitated the use of smaller units compared with those used for White recipients, which results in inferior outcome. UCB expansion technologies allow for the use of smaller, better matched units with the aim of improving outcome.”

In terms of end points measuring survival and incidences of graft versus host disease, there were no statistically significant differences reported in the study between the 2 arms. The investigators noted that while progression-free survival and overall survival trended higher in patients treated with omidubicel, they could not determine a statistically significant difference in these end points compared to the control arm due to the limited sample size of the study. They also noted that further investigation is needed to determine the performance of omidubicel after reduced intensity conditioning, and that future studies also ought to compare outcomes to other graft sources and specific disease types.

Despite these limitations, Horwitz and colleagues concluded that omidubicel may be considered as a new standard of care for adult patients eligible for UCBT.

1. Gamida Cell announces FDA acceptance of Biologics License Application for omidubicel with priority review. News release. Gamida Cell, Ltd. August 1, 2022. 
2. Horwitz ME, Stiff PJ, Cutler C, et al. Omidubicel vs standard myeloablative umbilical cord blood transplantation: results of a phase 3 randomized study. Blood. 2021;138(16):1429-1440. doi:10.1182/blood.2021011719
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