The FDA approved omidubicel on December 8, 2025, the same day parent company Ayrmid announced positive results at the 2025 American Society of Hematology Annual Meeting.
On December 8, 2025, the FDA approved omidubicel-onlv (Omisirge; Gamida Cell Ltd.), marking the first hematopoietic stem cell transplant (HSCT) therapy authorized to treat patients with severe aplastic anemia (SAA). The therapy is now approved for adults and pediatric patients 6 years and older following reduced-intensity conditioning when no compatible donor is available, according to a press release from the FDA. Additionally, omidubicel-onlv is indicated for adults and pediatric patients 12 years and older with hematologic malignancies.1
SAA is a rare and life-threatening disease in which the bone marrow fails to produce adequate red blood cells, white blood cells, and platelets. Standard treatment depends on age and often includes immunosuppressive therapy or hematopoietic stem cell transplant from a matched sibling or related donor. When a donor cannot be identified, a umbilical cord blood transplant may be used; however, this approach is limited by slow hematopoietic recovery and elevated infection risks.
Omidubicel-onlv is derived from donated cord blood stem cells that have been chemically enhanced with nicotinamide before administration to help restore blood and immune function. The therapy aims to overcome key limitations of umbilical cord blood transplantation, offering an additional graft option for patients with severe aplastic anemia who lack matched donors.1
“[Omidubicel-onlv] 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, MS, acting deputy director of the CBER Office of Therapeutic Products and pediatric hematologist, in a statement. "[Omidubicel-onlv] will shorten time to neutrophil recovery, which leads to shorter recovery times after transplant and may improve infection rates in this patient population.”
In the FDA-reviewed clinical study, omidubicel-onlv produced early and sustained neutrophil engraftment in 12 of 14 patients, with a median time to recovery of 11 days (range seven to 20 days). Reported adverse events included febrile neutropenia, viral and bacterial infections, hyperglycemia, immune thrombocytopenia, and pneumonia. Autoimmune cytopenias occurred in 25% of patients.
The FDA stated the open-label, prospective, single-arm study is ongoing in patients aged 6 years and older.
New interim clinical data shared at the 2025 American Society of Hematology (ASH) Annual Meeting further support the potential of omidubicel-onlv as a transplant option for severe aplastic anemia.2
In the ongoing open-label, single-center study of 19 patients whose disease had not responded to standard therapies, 95% achieved rapid neutrophil recovery, with a median time of eight days. Disease-free and overall survival were both 94%. Rates of graft-versus-host disease (GVHD) were low, with only 16% experiencing Grade II acute GVHD and no cases of severe or chronic GVHD reported.
Investigators called the results highly encouraging, emphasizing rapid engraftment, low incidence of mild acute GVHD, and the potential for a rapid return to normal life.
“The results of the ongoing study are extremely encouraging and indicate a significant advancement in the treatment options for patients with a high unmet medical need," said Richard Childs, MD, in a press release from Gamida. Childs is from the National Heart, Lung, and Blood Institute at the National Institutes of Health.
"The SAA patients in the study were high risk but had significantly better than expected outcomes with remarkably fast and high rates of neutrophil engraftment. This was achieved with low rates of mild acute GVHD and no chronic GVHD, meaning patients experienced a rapid return to normal life," added Childs.
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