FDA Approves First Cellular Therapy to Treat Patients with Severe Aplastic Anemia

Release date: 2025-12-10 10:15:50     Recommended: 92

The U.S. Food and Drug Administration (FDA) announced today that it has approved Omisirge (omidubicel-onlv), the first hematopoietic stem cell transplantation (HSCT) therapy for the treatment of patients with severe aplastic anemia (SAA). Originally approved for adult and pediatric patients aged 12 years and older with hematologic malignancies, Omisirge is now approved for adult and pediatric patients aged 6 years and older with SAA who are undergoing reduced-intensity conditioning and cannot find a compatible donor.

Expert Comments

Vinay Prasad, MD, MPH, Chief Medical and Scientific Officer and Director of the FDA’s Center for Biologics Evaluation and Research (CBER), stated: "This approval is revolutionary in the treatment field and fundamentally changes the way we treat SAA. Early treatment has the potential to alter the trajectory of patients' lives. Severe aplastic anemia is a potentially fatal rare blood disorder, and the FDA remains committed to expanding treatment options for patients living with this disease."

Megha Kaushal, MD, MSc, Acting Deputy Director of CBER’s Office of Therapeutic Products and a pediatric hematologist, commented: "Omisirge is a novel umbilical cord blood-derived stem cell product that will provide a treatment option for patients with severe aplastic anemia who have limited hematopoietic stem cell transplantation choices. Omisirge will shorten the time to neutrophil recovery, thereby reducing the post-transplant recovery period and potentially improving infection rates in this patient population."

About SAA

SAA is a rare and life-threatening blood disorder in which the bone marrow fails to produce sufficient red blood cells, white blood cells, and platelets. Treatment for SAA depends on age and typically includes immunosuppressive therapy and/or hematopoietic stem cell transplantation, with priority given to transplants from matched siblings or matched related donors. In the absence of a suitable donor, physicians may seek to treat SAA using umbilical cord blood transplantation. Umbilical cord blood transplantation is often limited by delayed hematopoietic recovery and increased risk of infection. Omisirge is a stem cell therapy in which donated umbilical cord blood stem cells are chemically enhanced with nicotinamide (a form of vitamin B3) and then infused into patients to help restore their blood and immune systems.

About Omisirge

Omisirge addresses the limitations of umbilical cord blood (UCB) as a stem cell source, including delayed hematopoietic recovery and increased infections, providing an additional graft option for patients with severe aplastic anemia (SAA) in need of hematopoietic stem cell transplantation (HSCT).

The safety and effectiveness of Omisirge were evaluated based on an ongoing, open-label, prospective, single-arm study assessing its use in patients with severe aplastic anemia aged 6 years and older. Among the 14 patients in the efficacy population, 12 achieved early and sustained neutrophil engraftment with Omisirge, with a median time to neutrophil recovery of 11 days (range: 7 to 20 days).

The most common side effects associated with Omisirge include febrile neutropenia, viral and bacterial infections, hyperglycemia, immune thrombocytopenia, and pneumonia. Autoimmune cytopenias occurred in 25% of patients.