FDA Accepts Priority Review Application of Omidubicel for Severe Aplastic Anemia

The US Food and Drug Administration (FDA) has accepted the priority review application for omidubicel, an investigative cell therapy, for the treatment of severe aplastic anemia (SAA).1

Announced by Ayrmid Ltd., parent company of cell therapy developer Gamida Cell Inc., the FDA has also assigned a Prescription Drug User Fee Act (PDUFA) target action date of December 10, 2025. The supplementary Biologics License Application submission is based on an investigator-sponsored study from the National Heart, Lung, and Blood Institute of the National Institutes of Health in London.1

“Priority review of omidubicel brings us closer to being able to provide a breakthrough orphan, potentially curative treatment for Severe Aplastic Anemia,” Joe Wiley, DPM, MBA, chairman and chief executive officer of Ayrmid Ltd., said in a statement. “We have the commercial and medical infrastructure in place to launch this therapy for patients suffering with this rare condition, and we look forward to sharing the exciting clinical data at upcoming medical meetings.”1

Omidubicel, a nicotinamide modified allogeneic hematopoietic progenitor cell therapy derived from umbilical cord blood, was initially launched in 2023 under the brand name Omisirge. Its initial indication was for adults and pediatric patients ≥12 years with hematologic malignancies who were planned for umbilical cord blood transplantation following myeloablative conditioning to reduce time to neutrophil recovery.1,2

SAA is a rare, life-threatening hematologic disease, typically characterized by low blood cell circulation and bone marrow failure. Although hematopoietic stem cell transplant may cure the disease, there is still a significant unmet need for patients without an available matched sibling donor.1

SAA is a largely acquired immune-mediated bone marrow failure disorder, manifesting in roughly 2 or 3 individuals per million. Its contributing factors are still not fully understood, but clinicians believe the condition is mediated by a combination of immune dysregulation and autoreactive cytotoxic T cell destruction of hematopoietic stem and progenitor cells (HSPC).3

The most common triggers of aplastic anemia include inherited bone marrow failure syndromes such as Fanconi anemia (a DNA repair disorder), dyskeratosis congenita (a telomere biology disorder), and the ribosome biology disorders Diamond Blackfan anemia and Shwachman Diamond syndrome. Additionally, many have associated congenital abnormalities and are often considered childhood diseases.3

Despite this, investigators have recently recognized that inherited bone marrow failure syndromes can occur without typical physical findings and/or family history, which can lead to delayed or outright misdiagnosis.3

Hematopoietic cell transplant is the recommended first-line therapy for patients <40 years with acquired SAA and a matched sibling donor. The 3-year survival probabilities for acquired SAA hematopoietic cell transplants are 90% for matched sibling donors and 76% for unrelated donors.3

If approved by the FDA, omidubicel may fill the significant unmet need for patients without matched sibling donors.1

References

1. Ayrmid Ltd. Ayrmid Ltd. Announces FDA Acceptance and Priority Review for Omidubicel for the Treatment of Severe Aplastic Anemia (SAA). BusinessWire. August 25, 2025. Accessed August 25, 2025. https://www.businesswire.com/news/home/20250822740040/en/Ayrmid-Ltd.-Announces-FDA-Acceptance-and-Priority-Review-for-Omidubicel-for-the-Treatment-of-Severe-Aplastic-Anemia-SAA

2. Morse AL, Kurz H, Moore DC. Omidubicel for Hematopoietic Cell Transplants: Considerations for Patients and Treatment Outcomes. Patient Prefer Adherence. 2024;18:2217-2223. Published 2024 Nov 5. doi:10.2147/PPA.S419253

3. McReynolds LJ, Rafati M, Wang Y, et al. Genetic testing in severe aplastic anemia is required for optimal hematopoietic cell transplant outcomes. Blood. 2022;140(8):909-921. doi:10.1182/blood.2022016508