Severe aplastic anaemia (SAA) leaves patients with almost no functioning bone marrow. Treatment often depends on finding a matched donor, which is not always possible. The FDA has now approved a breakthrough that changes this equation.
The US FDA has approved Omisirge (omidubicel-onlv) as the first hematopoietic stem cell transplant (HSCT) therapy for patients with severe aplastic anaemia. It is now cleared for adults and children aged six and older who need HSCT after reduced intensity conditioning and do not have a compatible donor.
Gamida Cell Ltd received the approval.
FDA leaders called this a “revolutionary” moment for the treatment landscape. Earlier treatment may help change long-term outcomes for many patients.
SAA is a rare and life-threatening disorder. Bone marrow fails to produce enough red cells, white cells, and platelets. Many patients rely on immunosuppressive therapy or donor-matched stem cell transplant. When matches are unavailable, umbilical cord blood (UCB) is often used.
However, UCB brings challenges:
Omisirge improves this process. It uses cord blood stem cells enhanced with nicotinamide, a form of vitamin B3. These modified cells help the immune and blood systems recover faster.
FDA experts highlighted that Omisirge shortens neutrophil recovery time. Faster recovery reduces infection risks and improves post-transplant outcomes.
In an ongoing clinical study:
This speed is a major improvement compared to traditional UCB transplants.
Common side effects include:
Autoimmune cytopenias occurred in 25% of patients.
The FDA granted Omisirge Orphan Drug and Priority Review designations, strengthening support for therapies addressing rare conditions.
Omisirge expands life-saving options for patients who previously had very few. Its approval signals a wider shift toward engineered stem cell therapies that shorten recovery times and broaden transplant eligibility.
For patients and caregivers navigating the challenges of SAA, this is a long-awaited advancement with real-world impact.
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