Ayrmid Ltd., parent company of cell therapy pioneer Gamida Cell Inc., announced that the U.S. Food and Drug Administration has accepted the company's priority review application for omidubicel as a treatment for severe aplastic anemia (SAA). The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of December 10, 2025, for this supplementary Biologics License Application.
The application is based on results from an investigator-sponsored study conducted at the National Heart, Lung, and Blood Institute of the National Institutes of Health. This represents a potential expansion of omidubicel's therapeutic applications beyond its current approved indication.
Addressing Critical Unmet Medical Need
Severe aplastic anemia is a rare, life-threatening hematologic disease characterized by bone marrow failure and low circulating blood cells. The condition affects approximately 2 to 3 individuals per million and is largely an acquired immune-mediated bone marrow failure disorder. While hematopoietic stem cell transplant may be curative for SAA, there remains a significant unmet need for patients who do not have an available matched sibling donor.
Current treatment outcomes show that hematopoietic cell transplant is the recommended first-line therapy for patients under 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.
Omidubicel's Therapeutic Profile
Omidubicel is a nicotinamide modified allogeneic hematopoietic progenitor cell therapy derived from umbilical cord blood. The therapy was initially launched in 2023 under the brand name Omisirge, with an approved indication for adults and pediatric patients 12 years and older with hematologic malignancies who were planned for umbilical cord blood transplantation following myeloablative conditioning to reduce time to neutrophil recovery.
The company's proprietary nicotinamide (NAM) technology leverages the properties of NAM to enhance and expand cells, creating allogeneic cell therapy products that are potentially curative for patients with hematologic conditions.
Commercial Readiness and Future Outlook
"Priority review of omidubicel brings us closer to being able to provide a breakthrough orphan, potentially curative treatment for Severe Aplastic Anemia," stated Dr. Joe Wiley, Chairman and Chief Executive Officer of Ayrmid Ltd. "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."
If approved, this new indication would complement Ayrmid's existing commercial hematology portfolio, which includes Omisirge for hematologic malignancies and APHEXDA for autologous transplantation. The potential approval could address the critical gap in treatment options for SAA patients without matched sibling donors, offering a potentially curative therapeutic alternative for this vulnerable patient population.
