The FDA and Gamida Cell have reached a consensus on the comparability of omidubicel manufactured at different sites.
Gamida Cell is planning to complete their biologics license application (BLA) for omidubicel (NiCord), a cell therapy for bone marrow transplant in patients with hematologic malignancies, in the first half of 2022 following positive Type B meeting correspondence from the FDA.1
“We are very pleased that our productive interactions with the FDA have resulted in alignment on the omidubicel manufacturing comparability analysis and agreement to initiate a rolling submission of our BLA application,” Julian Adams, PhD, chief executive officer, Gamida Cell, said in a statement.1 “Omidubicel is the first bone marrow transplant product to receive Breakthrough Therapy Designation from the FDA and has the potential to be the first FDA-approved advanced cell therapy for allogeneic bone marrow transplant. Initiating the BLA submission will move us one step closer toward bringing potentially curative therapies to patients. We plan to complete the full BLA submission in the first half of this year, which will be an important achievement for Gamida Cell and the bone marrow transplant community.”
Gamida Cell and the FDA reached an agreement on comparability of omidubicel manufactured at Gamida Cell’s own facility to the product manufactured at clinical manufacturing sites for the phase 3 study (NCT02730299). Gamida Cell had previously submitted a revised analysis of manufacturing data at the FDA’s request.2 The FDA did not request additional clinical data. Gamida Cell is initiating a rolling BLA submission which will be completed in the first half of 2022.
Omidubicel is an allogeneic hematopoietic cell therapy for bone marrow transplant in patients with leukemia, lymphoma, and myelodysplastic syndrome (MDS) developed using Gamida Cell’s proprietary nicotinamide-enabled cell expansion technology. It previously received breakthrough therapy designation from the FDA.
Omidubicel demonstrated efficacy and safety in patients with these conditions in the phase 3 study, data on which were published in Blood in June 2021.3The therapy yielded faster neutrophil and platelet engraftment than standard umbilical cord blood transplantation (UCBT), as well as fewer early bacterial and viral infections and less time in hospital.
Lead author Mitchell Horwitz, MD, professor of medicine, Duke Cancer Institute, and colleagues, found that median time to neutrophil engraftment—the primary end point of the study—was 12 days (95% CI, 10-14) in the omidubicel arm compared with 22 days (95% CI, 19-25) in the control arm (P <.001). Patients treated with omidubicel also had a 55% platelet recovery by 42 days post-transplant compared with a 35% recovery in the placebo group (P = .028).
“Previous studies have shown that engraftment with omidubicel is durable, with some patients in the Phase 1/2 study now a decade past their transplant. The Phase 3 data reinforce omidubicel’s potential to be a new standard of care for patients who are in need of stem cell transplantation but do not have access to an appropriate matched donor,” Horwitz said in a previous statement.3
Omidubicel had a good safety profile compared to standard UCBT, with treated patients having half the risk of grade 2 and 3 infections compared with UCBT (risk ratio, 0.5; P <.001). No statistically significant differences were observed in incidence of grade 3/4 acute GvHD between omidubicel (14%) and control (21%) arms or incidence of all grades of chronic GvHD at 1 year (omidubicel, 35%; control, 29%). Non-relapse mortality was 11% among those in the omidubicel group versus 24% for those in the control group (P = .09).