Omidubicel-onlv Gets Greenlight to Accelerate Neutrophil Recovery After Stem Cell Transplant in Hematologic Malignancies

FDA approves omidubicel for neutrophil recovery.

The FDA approved omidubicel-onlv (Omisirge), a substantially modified allogeneic cord blood-based cell therapy, for the treatment of patients aged 12 years and older with blood cancers who are planned for umbilical cord blood transplantation following a myeloablative conditioning regimen. The agent is designed to ramp up the recovery of neutrophils in the body and reduce the risk of infection.¹

Of note, the agent comes with a box warning for infisuion reactions, graft-vs-host-disease (GVHD), engraftment syndrome (identified as noninfectious fever and rash), and graft failure.¹

Findings from the phase 3 study (NCT02730299) showed that 87% of the 52 patients who received omidubicel achieved neutrophil recovery with a median time to recovery of 12 days (95% CI, 10-14). Patients in the control arm, who received the comparator treatment of umbilical cord blood transplantation (n = 56), experienced a neutrophil recovery rate of 83%—with a median time to recovery of 22 days (95% CI, 19-25; P < .001). Additionally, bacterial or fungal infections by 100 days following transplantation were seen in 39% vs 60% of patients in the investigational and control arm, respectively.^1,2

“Today’s approval is an important advance in cell therapy treatment in patients with blood cancers,” Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research said in a news release.¹ “Hastening the return of the body’s white blood cells can reduce the possibility of serious or overwhelming infection associated with stem cell transplantation. This approval reflects the FDA’s continued commitment to supporting development of innovative therapies for life-threatening cancers.”

Omidubicel is a cellular therapy that is patient-specific comprising human allogeneic stem cells from umbilical cord blood that are processed and cultured with nicotinamide. Each dose consists of healthy stem cells from an allogeneic prescreened donor. The agent is administered intravenously.^1,2

The multicenter study randomly assigned a total of 125 patients with confirmed blood cancers between the ages of 12 and 65 years to receive either omidubicel or standard umbilical cord blood transplantation. Patients had to be candidates for myeloablative allogeneic hematopoietic stem cell transplant and have no readily available matched donor to be included in the trial. Those with chronic lymphocytic leukemia or bone marrow fibrosis of 3+ were excluded.²

The primary end point was time to neutrophil engraftment. Secondary end points included platelet engraftment by 42 days, incidence of grade 2 to 3 bacterial or invasive fungal infection during the first 100 days, and days alive and out of the hospital for the first 100 days after transplantation.²

In terms of safety, results from the trial showed the most common treatment-emergent adverse effects (TEAEs) related to cord blood infusion included GVHD (35%), pain (8%), hypertension (4%), transplant failure (4%), and dyspnea (2%) for patients who received omidubicel. Patients who were treated with standard umbilical cord blood transplantation experienced these TEAEs at a rate of 25%, 2%, 18%, 9%, and 7%, respectively.²

Common grade 3 to 5 TEAEs in the investigational arm consisted of pain (32.7%), mucosal inflammation (30.8%), and hypertension (25.0%). In the control arm, TEAEs of grade 3 to 5 severity included hypertension (39.3%), gastrointestinal toxicity (33.9%), and mucosal inflammation (33.9%).²

References

1. FDA approves cell therapy for patients with blood cancers to reduce risk of infection following stem cell transplantation. News release. April 17, 2023. Accessed April 17, 2023. https://www.fda.gov/news-events/press-announcements/fda-approves-cell-therapy-patients-blood-cancers-reduce-risk-infection-following-stem-cell?utm_medium=email&utm_source=govdelivery
2. Horwitz ME, Stiff PJ, Cutler C, et al. Omidubicel vs standard myeloablative umbilical cord blood transplantation: results of a phase 3 randomized study. Blood. 2021;138(16):1429-1440. doi:10.1182/blood.2021011719