FDA Approves First Stem Cell Therapy for Severe Aplastic Anemia

FRIDAY, Dec. 12, 2025 (HealthDay News) — The U.S. Food and Drug Administration has approved Omisirge (omidubicel-onlv) as the first hematopoietic stem cell transplant therapy to treat patients with severe aplastic anemia (SAA).

Omisirge is now approved for adults and pediatric patients 6years of age and older with SAA following reduced intensity conditioning and for whom a compatible donor is not available.  

Omisirge is a stem cell therapy in which donated cord blood stem cells have been chemically enhanced with nicotinamide and then given to a patient to help restore their blood and immune system. The approval is based on a single-arm, open-label study that showed Omisirge provided early and sustained neutrophil engraftment in 12 of 14 patients, with a median time to neutrophil recovery of 11 days (range, seven to 20 days). The most common reported side effects include febrile neutropenia, viral and bacterial infections, hyperglycemia, immune thrombocytopenia, and pneumonia. One-fourth of patients had autoimmune cytopenias occur.  

“Omisirge will shorten time to neutrophil recovery which leads to shorter recovery times after transplant and may improve infection rates in this patient population,” Megha Kaushal, M.D., from the FDA Center for Biologics Evaluation and Research, said in a statement.

Approval of Omisirge was granted to Gamida Cell Ltd.

More Information