The FDA has approved omidubicel-onlv (Omisirge, Gamida Cell Ltd.) as the first hematopoietic stem cell transplant therapy for patients with severe aplastic anemia (SAA).

Specifically, omidubicel is approved for use following reduced-intensity conditioning in adult and pediatric patients aged 6 years or older for whom a compatible stem cell donor is not available, the FDA said.

The approval marks the second US indication for the novel blood-based cell therapy; omidubicel was first approved in 2023 for patients aged 12 years or older with hematologic malignancies who are scheduled for umbilical cord blood transplantation, to reduce infection risk and time to neutrophil recovery following myeloablative conditioning.

The latest approval, which was granted following priority review, was based on findings from an ongoing open-label, single-center, phase 1/2 study of patients with SAA — a rare, life-threatening hematologic disorder in which the bone marrow fails to produce sufficient blood cells. Omidubicel provided early and sustained neutrophil engraftment in 12 of 14 patients in the efficacy population, with a median time to neutrophil recovery of 11 days, the FDA said.

Updated findings presented on December 8 at the American Society of Hematology annual meeting showed that among 19 patients with a median age of 20 years, 18 (95%) achieved rapid neutrophil recovery within a median of 8 days. The disease-free and overall survival rates were both 94%, and no cases of severe acute or chronic graft-vs-host disease (GVHD) were observed, according to a press release from Ayrmid Ltd., the parent company of Gamida Cell Inc.

Stem cell transplantation offers a potential cure for SAA, but many patients lack a matched sibling donor. Omidubicel provides an alternative treatment for these patients, the company said.

“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,” lead investigator Richard Childs, MD, of the National Heart, Lung, and Blood Institute, stated in the Ayrmid press release.

“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,” Childs said. The lack of severe GVHD, he noted, meant that patients “achieved a rapid return to a normal life.”

According to the FDA, the most common side effects associated with omidubicel include febrile neutropenia, viral and bacterial infections, hyperglycemia, immune thrombocytopenia, and pneumonia. Autoimmune cytopenias have occurred in 25% of patients.

Full prescribing information will be available at the FDA.gov website.

Sharon Worcester, MA, is an award-winning medical journalist based in Birmingham, Alabama, writing for Medscape Medical News, MDedge, and other affiliate sites. She currently covers oncology, but she has also written on a variety of other medical specialties and healthcare topics. She can be reached at sworcester@mdedge.comor on X @SW_MedReporter.