What's Happening?
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.
Why It's Important?
The approval of Omisirge is significant as it provides a new treatment option for SAA patients without a family donor, who previously had limited options. This therapy offers a potential cure with high survival rates and manageable toxicity, reducing treatment time from 25 days to eight days compared to traditional cord blood procedures. The development is a breakthrough in the therapeutic landscape, fundamentally changing the approach to treating SAA. It highlights the FDA's commitment to advancing innovative treatments for rare diseases, potentially improving the quality of life for affected patients and reducing healthcare costs associated with long-term care.
What's Next?
Following the FDA approval, Gamida Cell is expected to focus on the commercial rollout of Omisirge, ensuring accessibility to patients in need. The company may also continue to monitor the long-term efficacy and safety of the therapy in real-world settings. Healthcare providers and insurers will likely evaluate the cost-effectiveness of Omisirge compared to existing treatments. Additionally, the approval may encourage further research and development in cell therapies for other rare hematologic disorders, potentially leading to more breakthroughs in the field.
