New data presented at the 2025 Association for the Advancement of Blood & Biotherapies (AABB) Annual Meeting highlights positive outcomes for patients
Reports 92.9% of patients achieved rapid neutrophil recovery, with median time to neutrophil recovery of 7 days
Prepares for Prescription Drug User Fee Act (PDUFA) target action date of December 10, 2025
LONDON--(BUSINESS WIRE)--Ayrmid Ltd. (“Ayrmid” or the “Company”), the parent company of Gamida Cell Inc., a cell therapy pioneer working to turn cells into powerful therapeutics, announced positive interim study results for omidubicel, a stem cell transplant therapy, in treating Severe Aplastic Anemia (SAA). The findings were shared by Dr. Ronit Simantov, Chief Medical and Scientific Officer of Gamida Cell, during a presentation at the 2025 Association for the Advancement of Blood & Biotherapies (AABB) Annual Meeting.
Interim results from the study, led by Dr. Richard Childs of the National Heart, Lung, and Blood Institute (NHLBI) at the National Institutes of Health (NIH), were highly encouraging, with 13 of the 14 patients (92.9%) reaching rapid neutrophil recovery, with a median recovery time of 7 days. The disease-free survival rate, as well as overall survival, was 92.3%, comparing favorably with outcomes from patients who receive transplants from matched donors. Additionally, no severe (Grade III–IV) graft-versus-host disease (GvHD) or chronic GvHD was observed, and only 14% of patients experienced moderate (Grade II) GvHD.
Dr. Simantov commented: “Gamida Cell has long been dedicated to developing breakthrough therapies for patients with significant unmet medical needs. The results of this study demonstrate omidubicel, with a 92.3% disease-free survival, can offer a real option for patients with severe aplastic anemia who do not have a matched donor, expanding curative transplant access for patients. We are focused on working towards the FDA’s priority review to bring this therapy to patients and their physicians with a PDUFA target date of December 10, 2025.”
The open-label, single-center study was designed to evaluate whether a single transplanted omidubicel unit could achieve rapid and durable blood cell recovery in patients with Severe Aplastic Anemia who lacked a matched donor. The study of omidubicel transplantation used a reduced intensity conditioning regimen and included 14 patients (median age 22 years) whose diseases had not responded to standard therapies.
This study, supplemented with additional data, forms the basis for the company’s submission to the U.S. Food and Drug Administration (FDA) for approval with a Prescription Drug User Fee Act (“PDUFA”) target action date of December 10, 2025.
About Severe Aplastic Anemia
Severe Aplastic Anemia (SAA) is a rare, life-threatening hematologic disease characterized by bone marrow failure and low circulating blood cells. Bone marrow stem cell transplant may be curative for SAA, but there is a significant unmet need for patients who do not have an available matched sibling donor. Gamida Cell is advancing omidubicel to help bridge that gap.
About Ayrmid Ltd. and Gamida Cell
Ayrmid Ltd. is the parent company of Gamida Cell Inc., a cell therapy pioneer working 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
Investors / Business Development
bd@ayrmid.com
