FDA Approves Omisirge for Severe Aplastic Anaemia, Offering New Treatment Options
The FDA has approved Omisirge, a cell therapy developed by Gamida Cell, for the treatment of severe aplastic anaemia (SAA) in patients aged six and over who do not have a matched haematopoietic stem cell transplant (HSCT) donor. This approval follows positive results from a pivotal trial presented at the American Society of Hematology (ASH) congress. Omisirge, which has been used since 2023 to reduce infection risk in blood cancer patients undergoing HSCT, is now extended to treat SAA, a rare and life-threatening disorder where the bone marrow fails to produce enough blood cells. The therapy is based on cord blood stem cells enhanced with nicotinamide, a form of vitamin B3, to increase cell quantity and functionality. In trials, 95% of patients achieved rapid recovery of infection-fighting neutrophil cells, with a median recovery time of eight days. 
