FDA Manufacturing Data Request Delays Omidubicel BLA Submission


The request aims to ensure the comparability of omidubicel manufactured at different sites.

The FDA has requested a revised analysis of manufacturing data from Gamida Cell on omidubicel (NiCord), a cell therapy for bone marrow transplant in patients with hematologic malignancies. Following their meeting, Gamida Cell expects to submit a biologics license application (BLA) in early 2022 as opposed to late 2021.1

The FDA requested the additional data to ensure the comparability of omidubicel manufactured at Gamida Cell’s own facility to the product manufactured at clinical manufacturing sites for the phase 3 study (NCT02730299). The FDA did not request additional clinical data.

“Despite the delay in timing to bring omidubicel to patients after a potential FDA approval, we are encouraged by the FDA’s reaction to our Phase 3 data as the pivotal trial of omidubicel. We have gained further clarity with the FDA on the requirements for demonstrating comparability for our commercial manufacturing facility,” Julian Adams, PhD, chief executive officer, Gamida Cell, said in a statement.1 “With the FDA’s feedback in hand, we believe that we are one step closer for omidubicel to be made available to patients in need.”

Omidubicel, which previously received breakthrough therapy designation from the FDA, is a cell therapy for bone marrow transplant in patients with leukemia, lymphoma, and myelodysplastic syndrome (MDS). It is developed using Gamida Cell’s proprietary nicotinamide-enabled cell expansion technology.

WATCH NOW: Improving Bone Marrow Transplant With Omidubicel

Omidubicel is being evaluated in a phase 3 study. Updated results from the trial were published in Blood in June 2021 and showed that the therapy yielded faster neutrophil and platelet engraftment, as well as fewer early bacterial and viral infections and less time in hospital compared with standard umbilical cord blood transplantation (UCBT).2

Investigators, including 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). In addition, patients treated with omidubicel 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

In terms of safety, analysis revealed that patients receiving omidubicel had half the risk of grade 2 and 3 infections compared with those who received standard 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).

1. Gamida Cell Provides Update on Pre-BLA Meeting With FDA for Omidubicel. News release. Gamida Cell. News release. November 11, 2021. https://www.businesswire.com/news/home/20211111005505/en/Gamida-Cell-Provides-Update-on-Pre-BLA-Meeting-With-FDA-for-Omidubicel
1. Horwitz ME, Stiff PJ, Cutler CS, et al. Omidubicel versus standard myeloablative umbilical cord blood transplantation: Results of a phase III randomized study. Blood. Published online June 22, 2021. doi: 10.1182/blood.2021011719
3. Gamida Cell announces publication in Blood, the journal of the American Society of Hematology, of the first pivotal trial to evaluate a cell therapy (omidubicel) for patients with blood cancer who require an allogeneic stem cell transplant. News release. Gamida Cell. June 23, 2021. https://www.businesswire.com/news/home/20210623005336/en/Gamida-Cell-Announces-Publication-in-Blood-the-Journal-of-the-American-Society-of-Hematology-of-the-First-Pivotal-Trial-to-Evaluate-a-Cell-Therapy-Omidubicel-for-Patients-with-Blood-Cancer-who-Require-an-Allogeneic-Stem-Cell-Transplant
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