The US Food and Drug Administration (FDA) has approved omidubicel-onlv, which is intended for use in patients 12 years and older with blood cancers who planned to receive umbilical cord blood transplantation following a myeloablative conditioning regimen.
Omidubicel-onlv is a “substantially modified” allogeneic cord blood-based cell therapy to “quicken the recovery of neutrophils” and reduce the risk of infection, according to a news release from the FDA.
“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. “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-onlv is administered as a single intravenous dose and each dose is patient-specific.
Its safety and efficacy were supported by a randomized, multicenter study comparing transplantation of omidubicel-onlv to transplantation of umbilical cord blood in patients aged 12 to 65 years. All 125 patients in the study had confirmed blood cancers.
The study showed 87% of patients who received omidubicel-onlv achieved neutrophil recovery at a median of 12 days following treatment, while 83% of patients who received umbilical cord blood transplantation achieved neutrophil recovery with a median of 22 days.
At 100 days after transplantation, 39% of patients who received omidubicel-onlv had bacterial or fungal infections, while 60% of patients who received umbilical cord blood did.
The omidubicel-onlv label carries a Boxed Warning for infusion reactions, graft-versus-host disease, engraftment syndrome, and graft failure.
The application received Priority Review, Breakthrough Therapy, and Orphan Designations. The FDA granted regular approval of omidubicel-onlv to Gamida Cell Ltd.
Source: US FDA, April 2023