You are about to leave this website for another external website outside of the control of Gamida Cell. Gamida Cell has no responsibility for the content of such other sites and is not liable for any damages or injury arising from that content. Any links to other sites are provided merely as a convenience to the users of this website.
Additional Positive Results for Omidubicel in Treating Severe Aplastic Anemia Presented at ASH
Dec 8, 2025
New data presented at the 2025 ASH demonstrates 94% disease free and overall survival rate
Key Highlights of Data Presented at ASH:
- 95% of patients achieved rapid neutrophil recovery
- Median time to neutrophil recovery: 8 days
- Immune recovery post-transplant was rapid
- 94% disease-free and overall survival
- No cases of BMT-CTN severe acute graft-versus-host disease (GVHD), or chronic GVHD observed
- PDUFA target action date: December 10, 2025
LONDON – December 8, 2025 – Ayrmid Ltd. (“Ayrmid” or the “Company”), the parent company of Gamida Cell Inc., a leader in cell therapy innovation today announced further positive interim clinical results for omidubicel, its advanced stem cell transplant therapy for Severe Aplastic Anemia (SAA). The data were presented by Dr. George Aue of the National Heart, Lung, and Blood Institute (NHLBI) at the National Institutes of Health (NIH), at the American Society of Hematology (ASH) 2025 Annual Meeting, taking place from Dec 6-9 in Orlando FL.
The ongoing open-label, single-center study (NCT03173937 | 17-H-0091), led by Dr. Richard Childs of the National Heart, Lung, and Blood Institute (NHLBI) at the National Institutes of Health (NIH), demonstrated highly encouraging results. Among 19 patients (median age 20 years) whose disease had not responded to standard therapies, 18 (95%) achieved rapid neutrophil recovery with a median time of 8 days. Both disease-free survival and overall survival were 94%. Importantly, only 16% of patients experienced (BMT-CTN Grade II) acute GvHD and no cases of severe (BMT-CTN Grade III–IV) acute GVHD or chronic GVHD were observed.
Dr. Richard Childs of the National Heart, Lung, and Blood Institute (NHLBI) at the National Institutes of Health (NIH) commented: “The results of the ongoing study are extremely encouraging and indicate a significant advancement in the treatment options for patients with a high unmet medical need. The SAA patients in the study were high risk but had significantly better than expected outcomes with remarkably fast and high rates of neutrophil engraftment. This was achieved with low rates of mild acute GVHD and no chronic GVHD, meaning patients experienced a rapid return to normal life.”
Dr. Ronit Simantov, Chief Medical and Scientific Officer of Ayrmid commented: “These results highlight the significant potential of Omisirge® as a transplant option for patients with SAA given the rapid and sustained recovery of blood counts observed. Omisirge potentially expands transplant access for patients who otherwise have limited options. We remain deeply committed to advancing transformative therapies for patients with serious unmet medical needs. We look forward to progressing the FDA’s review process, with a PDUFA target date of December 10, 2025.”
This study of omidubicel with a reduced-intensity conditioning regimen, supports Gamida Cell’s submission to the U.S. Food and Drug Administration (FDA) for approval of OMISIRGE (Omidubicel-onlv) in SAA with a Prescription Drug Free User Act (“PDUFA”) target action date of December 10, 2025.
About Severe Aplastic Anemia
Severe Aplastic Anemia is a rare, life-threatening hematologic disorder in which the bone marrow fails to produce sufficient blood cells. Stem cell transplantation offers a potential cure; however, many patients lack a matched sibling donor. Ayrmid is advancing therapies to address this unmet medical need.
About Ayrmid Ltd. and Gamida Cell
Ayrmid Ltd. is the parent company of Gamida Cell Inc., a pioneering cell therapy company developing novel treatments designed to turn cells into powerful therapeutics. Gamida Cell Inc. currently has two FDA approved products on the market in the US, namely Omisirge (please see the current full Prescribing Information, including boxed warning, here) and APHEXDA® (please see the current full Prescribing Information here). Gamida Cell operates as a wholly owned subsidiary of Ayrmid Limited, a UK entity. For additional information, please visit www.gamida-cell.com or follow Gamida Cell on LinkedIn, X, Facebook or Instagram.
Contacts: Media, Investors / Business Development: bd@ayrmid.com
