Within 60 days, the FDA will make a decision on whether to accept the biologics license application for omidubicel as treatment for patients with blood cancers in need of an allogeneic hematopoietic stem cell transplant.
A rolling submission of a biologics license application has been submitted to the FDA seeking approval of omidubicel for the treatment of patients with blood cancers in need of an allogeneic hematopoietic stem cell transplant, according to an announcement by Gamisa Cell Ltd.
In a phase 3 study, treatment with omidubicel showed encouraging clinical benefit compared with standard myeloablative umbilical cord blood (UCB), leading investigators to conclude that this strategy may replace UCB in the future.
“The BLA submission marks an important milestone for both Gamida and the transplant community, as omidubicel has the potential to be the first approved advanced cell therapy product for allogeneic stem cell transplantation,” said Julian Adams, PhD, chief executive officer of Gamida Cell, in a press release. “Completion of this BLA submission is a key inflection point in our mission to deliver a new treatment option for patients with blood cancers. We look forward to working closely with the FDA to bring this potentially important therapy to patients.”
In the study of omidubicel vs UCB in patients with hematologic malignancies, the goal was to determine the efficacy of omidubicel. The study included patients had to be between the ages of 12 years and 65 years who had high-risk hematologic malignancies, were candidates for allogeneic bone marrow transplantation and did not have a matched donor.
Patients in the intent-to-treat (ITT) population were randomized to receive omidubicel (n = 62) vs standard UCB (n = 63). Of those patients randomized, 108 were classified as the as-treated (AT) population who received either omidubicel (n = 52) or standard UCB (n = 56). The patients were stratified by treatment center, disease risk index (DRI), age, and intent to perform a single or double cord transplant in the control arm.
The study assessed the primary end point of time to neutrophil engraftment, and secondary end points of time to platelet engraftment, the incidence of grades 2/3 invasive bacterial or fungal infections at 100 days, and days alive out of the hospital following the first 100 days post-transplantation.
At baseline, the patient demographics appeared well-balanced between the 2 treatment arms. Patients in the ITT population had a median age of participants 41.5 years and 57.5% were male. The majority of patients (48%) had acute myeloid leukemia, 32.5% had acute lymphocytic leukemia, 7.5% had myelodysplastic syndromes, 5% had chronic myeloid leukemia, 4% had lymphoma, and 3% had a rare leukemia. In terms of race, most patients identified as White (58%), 16% were Black, 13.5% were Asian, and 13% were other.
Results showed that the median time to neutrophil engraftment in the ITT population was 12 days (95% CI, 10-15) in the omidubicel ITT arm, and 22 days (95% CI, 19-25) in the control ITT arm (P <.001), achieving statistical significance.
In the AT population the median time to neutrophil recovery was 10 days (95% CI, 8-13) in the omidubicel arm vs 20.5 days (95% CI, 18-24) in the control arm (P <.001). The cumulative incidence of neutrophil engraftment was 96% in those who received omidubicel vs 89% with the control.
Platelet engraftment by day 42 was achieved in 55% of those in the ITT population who received omidubicel compared with 35% of those treated with the control. This translated to a 20% difference in cumulative incidence between the arms (P = .028). Further, the cumulative incidence of platelet engraftment in the AT population at day 100 was 83% at a median of 37 days (95% CI, 33-42) with omidubicel vs 73% at a median of 50 days (95% CI, 42-58) with UCB (P <.023), also achieving statistical significance.
In the ITT population, treatment with omidubicel correlated with a lower likeliness of developing grade 2/3 bacterial or invasive fungal infection by day 100. The incidence of these infections was 37% in the omidubicel arm vs 57% in UCB arm (P = .027).
The FDA has 60 days to decide whether the BLA for omidubicel seeking approval for the treatment of patients with blood cancers in need of an allogeneic hematopoietic stem cell transplant is acceptable for filing.
1. Gamida Cell completes rolling biologics license application submission to the FDA for omidubicel. News release. June 2, 2022. Accessed June 3, 2022. https://bwnews.pr/3mblBWN
2. Horwitz ME, Stiff P, Rezvani AR, et al. Improved clinical outcomes with omidubicel versus standard myeloablative umbilical cord blood transplantation: results of a phase III randomized, multicenter study. Presented at: 2021 Transplantation and Cellular Therapy Meetings; February 8-12, 2021; Virtual. Abstract 34.