FDA

First cell therapy approved for severe aplastic anemia

December 10, 2025

On December 8, 2025, FDA approved Omisirge (omidubicel-onlv), the first hematopoietic stem cell transplant (HSCT) therapy to treat patients with severe aplastic anemia (SAA). Omisirge is indicated for adults and pediatric patients ≥12 years of age with hematologic malignancies and now is approved for adults and pediatric patients ≥6 years of age with SAA following reduced intensity conditioning and for whom a compatible donor is not available.

Omisirge is a nicotinamide (NAM) modified allogeneic hematopoietic progenitor cell therapy derived from cord blood used as an allogeneic stem cell donor source.

Safety and effectiveness of Omisirge were assessed based on an ongoing, open-label, prospective, single arm study (NCT03173937) evaluating use of Omisirge in patients six years and older with severe aplastic anemia. Omisirge provided early and sustained neutrophil engraftment in 12 of 14 patients in the efficacy population with a median time to neutrophil recovery of 11 days (range, 7-20 days).

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

Sources:

FDA approves first cellular therapy to treat patients with severe aplastic anemia. [News release]. 2025. https://www.fda.gov/news-events/press-announcements/fda-approves-first-cellular-therapy-treat-patients-severe-aplastic-anemia

Omisirge (omidubicel-onlv) [package insert]. Gamida Cell. https://www.gamida-cell.com/wp-content/uploads/Omisirge-final-PI.pdf Revised December 2025. Accessed December 9, 2025.

FDA approves Omisirge® as first approved cell therapy to treat severe aplastic anemia. [News release]. 2025. https://www.gamida-cell.com/press_release/fda-approves-omisirge-as-first-approved-cell-therapy-to-treat-severe-aplastic-anemia/