FDA approves Omisirge(R) as First Approved Cell Therapy to Treat Severe Aplastic Anemia

Study demonstrated early and sustained hematopoietic recovery and transfusion independence

Omisirge is a new and novel transplant option for patients with SAA

Additional manufacturing capacity for Omisirge will be established in the U.S.

Represents a supplementary FDA approval for Omisirge

DUBLIN, IE AND LONDON, UK / ACCESS Newswire / December 5, 2025 / Ayrmid Ltd. (“Ayrmid” or the “Company”), the parent company of Gamida Cell Inc., a leader in cell therapy innovation announces that it has received U.S. Food and Drug Administration (“FDA”) approval for Omisirge (Omidubicel-onlv) for the treatment of Severe Aplastic Anemia (SAA).

This approval is based on the ongoing open-label, single-center study, led by Dr. Richard Childs of the National Heart, Lung, and Blood Institute (NHLBI) at the National Institutes of Health (NIH), which demonstrated highly encouraging results including:

• Median time to neutrophil recovery: 11 days

• 86% of patients had early and sustained neutrophil recovery at 100 days

• Immune recovery post-transplant was rapid

• 86% of patients achieved red blood cell transfusion independence

• No cases of BMT-CTN severe acute graft-versus-host disease (GVHD), or chronic GVHD observed

• 92% disease-free and overall survival rate

Dr. Richard Childs of the National Heart, Lung, and Blood Institute (NHLBI) at the National Institutes of Health (NIH) commented: “The approval of Omisirge is a significant step forward in the treatment options available for patients with a high unmet medical need. Patients in the study with aplastic anemia were high risk, but had significantly better than expected outcomes and demonstrated remarkably fast and high rates of neutrophil engraftment. This was achieved with low rates of mild acute GVHD and no chronic GVHD meaning the patients achieved a rapid return to a normal life.”

Dr. Joe Wiley, Ayrmid Chairman and Chief Executive Officer commented: “Alongside its initial FDA approval in hematological malignancy in 2023, Omisirge is a new and novel transplant option for the many SAA patients who do not have a matched related donor. This approval is potentially transformative for patients living with SAA, given the rapid and sustained recovery of blood counts observed. At Ayrmid, we remain deeply committed to advancing innovative therapies to help patients with serious unmet medical needs.”

To meet the expected demand from SAA patients, Ayrmid has a strategic partnership in place with RoslinCT for the production of Omisirge in the U.S. alongside existing manufacturing facilities. The technology transfer and commercial manufacturing of Omisirge will take place in RoslinCT’s state-of-the-art, purpose-built cell therapy CGMP manufacturing facility in Hopkinton, MA. beginning in 2027.

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

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SOURCE: Ayrmid Pharma Ltd.

View the original press release on ACCESS Newswire