FDA Approves Omidubicel to Reduce Infection Risk in Patients With Hematologic Malignancies Undergoing Stem Cell Transplant


Infection rates in patients receiving omidubicel post-stem cell transplant were about 20% less than those in the control group.

Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research

Peter Marks, MD, PhD

The FDA has approved Gamida Cell's allogeneic cell therapy, omidubicel, to reduce the risk of infection in patients aged 12 years and older undergoing stem cell transplantation for hematologic cancer.1 The approval marks a significant step forward in the cell therapy space as researchers continue to identify unmet treatment needs.

Omidubicel, which will be marketed as Omisirge, is delivered as a single intravenous infusion composed of patient-specific human allogeneic cord blood-derived stem cells that have been processed and cultured with nicotinomide, a form of vitamin B3.

“Today’s approval is an important advance in cell therapy treatment in patients with blood cancers. Hastening the return of the body’s white blood cells can reduce the possibility of serious or overwhelming infection associated with stem cell transplantation. This approval reflects the FDA’s continued commitment to supporting development of innovative therapies for life-threatening cancers,” Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, said in a statement.1

The efficacy of omidubicel has been demonstrated in an ongoing multicenter phase 3, open-label, controlled, randomized clinical trial in 125 participants who were eligible for allogeneic transplant and had acute lymphoblastic leukemia or acute myeloid leukemia, myelodysplastic syndrome, chronic myeloid leukemia, or lymphoma.

Chenyu Lin, MD, a hematology/oncology fellow at Duke Cancer Institute of Duke University, told CGTLive that, "I think this is a tremendous step forward for patients who are in need of an allogeneic stem cell transplantation for blood cancers. Many patients don't have an optimal stem cell donor, and omidubicel adds a new option to our armamentarium. In particular, those patients needing an umbilical cord blood transplant will have the benefit of using an approach with demonstrated safety. The studies have shown that omidubicel is associated with faster recovery of blood counts, a lower risk of infection, shorter hospital stays, and better quality of life compared to conventional cord blood transplants."

Lin added that the cord expansion technology with the therapy might allow smaller cord blood units to be used, with the aim that "this will expand the donor pool, increasing access to stem cell transplantation, particularly for our patients from racial minority backgrounds who may have a harder time finding donors."

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An analysis recently presented at the 2022 American Society of Hematology Annual Meeting comparing the efficacy of omidubicel to other allogeneic hematopoietic cell transplant donor sources in a real-world analysis showed that treatment with omidubicel was associated with more rapid neutrophil recovery compared with other donor sources (median, 10 days vs 15-20 days; P <.001). The cell therapy was also associated with slower platelet engraftment than the haploidentical, 8/8-matched unrelated (MUD), or 7/8-matched unrelated (MMUD) donor sources but faster platelet engraftment than unmanipulated cord blood (median, 35 days vs. 19-26 vs. 43 days; P <.001).2

Notably, rates of grade II-IV acute graft-versus-host disease (GvHD) in the first 100 days post-transplant were higher in those who received omidubicel (60% vs 32-43% for other sources); however, rates of grade III-IV GvHD over the first 100 days as well over the first 12 months were comparable between all donor source groups.

Additionally, data from omidubicel’s phase 3 trial presented at the 10th Annual Meeting of the Society of Hematologic Oncology (SOHO), which took place September 28-October 1, 2022 in Houston, Texas, showed an overall survival rate of 63% (95% CI, 53%-73%) and a disease-free survival rate of 56% (95% CI, 47%-67%) at a median follow-up of 22 months (range, 0.3-122.5) overall and 35.7 months (range, 11.7-122.5) for survivors among those treated with omidubicel.3

Omidubicel has previously been granted breakthrough therapy designation by the FDA and orphan drug designation by both the FDA and the European Medicines Agency. The therapy's biologics license application (BLA) review was extended in late 2022, more than 3 months after the company originally announced the acceptance for filing with priority review of omidubicel’s application by the FDA in August 2022.4

The extension was deemed related to laboratory data for intermediate time points from patients participating in the phase 3 clinical trial (NCT0273029) that Gamida Cell gave in response to a request for information issued by the FDA, which the agency considered a major amendment.4 The company noted that these laboratory results are consistent with data included in previous submissions. Gamida Cell’s late-cycle meeting with the FDA has additionally been rescheduled to the first quarter of next year.

1. FDA Approves Cell Therapy for Patients with Blood Cancers to Reduce Risk of Infection Following Stem Cell Transplantation. News release. FDA. April 17, 2023. Accessed April 17, 2023.https://www.fda.gov/news-events/press-announcements/fda-approves-cell-therapy-patients-blood-cancers-reduce-risk-infection-following-stem-cell
2. Sivaraman S, Sajeev G, Song Y, et al. Clinical Outcomes Following Allogeneic Hematopoietic Cell Transplantation with Omidubicel or Other Donor Sources in Patients with Hematologic Malignancies: Comparison of Clinical Trial Results to Center for International Blood and Marrow Transplant Research Database Controls. Blood. 2022;140(Suppl 1):660–661. doi:10.1182/blood-2022-162439
3. Long-term data from omidubicel phase 3 trial demonstrates overall survival and sustainable durable outcomes for patients with blood cancers at the Society of Hematologic Oncology Meeting. News release. Gamida Cell. September 29, 2022. Accessed April 17, 2023. https://www.businesswire.com/news/home/20220929005316/en/Long-Term-Data-from-Omidubicel-Phase-3-Trial-Demonstrates-Overall-Survival-and-Sustainable-Durable-Outcomes-for-Patients-with-Blood-Cancers-at-the-Society-of-Hematologic-Oncology-Meeting
4. Gamida Cell provides regulatory update on omidubicel. News release. Gamida Cell Ltd. News release. November 21, 2022. Accessed April 17, 2023. https://investors.gamida-cell.com/news-events/press-releases/news-release-details/gamida-cell-provides-regulatory-update-omidubicel
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