New Cord Blood Stem Cell Approval Alters Biopharma Manufacturing Priorities

FDA’s latest approval of omidubicel-onlv (brand name Omisirge) makes it the first regulated cellular therapy authorized to treat patients with severe aplastic anemia (SAA), the agency announced on Dec. 8, 2025 (1). The therapy is indicated for adults and pediatric patients six years and older with the disease who lack access to a compatible donor. This approval expands the therapy’s earlier use in patients with blood cancers following initial FDA approval in April 2023 (2).

The marketing authorization in this expanded indication was granted to Gamida Cell, a cell therapy company, and it adds a commercially manufactured, chemically enhanced cord blood product to the regulated transplant and cell therapy landscape (3). FDA’s decision also introduces a new category of chemically modified hematopoietic stem cell products into clinical practice, with implications for manufacturing standards, facility design, and regulatory expectations across the cell therapy sector (3).

“This approval is revolutionary in the therapeutic landscape and fundamentally changes how we approach treatment for SAA, where earlier treatment has potential to alter one’s life course,” said Vinay Prasad, MD, chief medical and scientific officer, FDA, and director of FDA’s Center for Biologics Evaluation and Research (CBER), in an agency press release (1). “Severe aplastic anemia is a rare blood disorder that can be fatal, and [FDA] remains committed to expanding treatment options for patients with this disease.”

What is severe aplastic anemia and what impact does this approval have?

SAA is a rare and life-threatening bone marrow failure disorder in which patients cannot produce adequate red blood cells, white blood cells, or platelets, according to FDA. “Treatment for SAA depends on age and usually consists of either immunosuppressive therapy and/or hematopoietic stem cell transplant preferably from a matched sibling or matched related donor,” the agency stated in the release (1). “If a donor is not available, providers may seek the use of umbilical cord transplant to treat SAA.” Umbilical cord transplant typically has limitations of use, however, including delayed hematopoietic recovery and increased risks of infections, the agency also stressed in the release (1).

Omidubicel-onlv is manufactured from donated umbilical cord blood that is chemically enhanced with nicotinamide, a form of vitamin B3, to improve the functional performance of transplanted stem cells (1). This ex-vivo manipulation step represents a technical shift away from minimally processed grafts and toward engineered cellular products, increasing the importance of controlled processing environments, closed-system manufacturing, and advanced in-process analytics (3,4).

This approval reinforces emerging expectations for scalable, standardized, and compliant platforms capable of chemically modifying living cells under stringent current good manufacturing practice conditions. The need to maintain cell potency while introducing chemical enhancement steps is likely to influence future investments in automation, single-use processing, and real-time release testing (5).

What does this decision mean for future rare-disease cell therapy development?

Omidubicel-onlv’s safety and effectiveness were evaluated in an ongoing, open-label, prospective, single-arm study enrolling patients aged six years and older with SAA. The therapy achieved early and sustained neutrophil engraftment in 12 of 14 patients, with a median time to neutrophil recovery of 11 days, ranging from seven to 20 days. These outcomes are relevant to industry because accelerated neutrophil recovery can reduce inpatient resource utilization and shift economic assumptions for complex cell therapies, according to FDA.

“Omisirge is a novel stem cell product from umbilical cord blood that will be able to offer a therapeutic option for patients with severe aplastic anemia who have limited options for stem cell transplant,” said Megha Kaushal, MD, acting deputy director of the CBER Office of Therapeutic Products and pediatric hematologist, in the release (1). “Omisirge will shorten time to neutrophil recovery which leads to shorter recovery times after transplant and may improve infection rates in this patient population.”

The most common adverse events included febrile neutropenia, viral and bacterial infections, hyperglycemia, immune thrombocytopenia, and pneumonia. Autoimmune cytopenias occurred in 25% of treated patients. The application for the therapy received orphan drug and priority review designations, which underscores regulatory willingness to accelerate review for high-need indications.

References

1. FDA. FDA Approves First Cellular Therapy to Treat Patients with Severe Aplastic Anemia. Press Release. Dec. 8, 2025.
2. FDA. FDA Approves Cell Therapy for Patients with Blood Cancers to Reduce Risk of Infection Following Stem Cell Transplantation. Press Release. April 17, 2023.
3. Horwitz, M. E.; Schiller, G. J.; Tsai, S. B.; et al. Omidubicel-onlv Transplantation for Hematologic Malignancies: Results of a Multicenter Expanded Access Program. Transplant. Cell. Ther. 2025, 31 (7), 436–447. DOI: 10.1016/j.jtct.2025.04.005
4. Horwitz, M. E.; Stiff, P. J.; 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
5. Jiang, M.; Severson, K. A.; Love, J. C.; et al. Opportunities and Challenges of Real-Time Release Testing in Biopharmaceutical Manufacturing. Biotechnol. Bioeng. 2017, 114 (11), 2445–2456. DOI: 10.1002/bit.26383