FDA-Approved Cell Therapy Protects Patients After Stem Cell Transplant

The FDA recently approved Omisirge (omidubicel-onlv), a new cell therapy meant to protect patients with blood cancer after a stem cell transplant. According to the press release, the treatment can minimize the risk of infection for these patients.

The treatment involves an infusion of modified donor cord blood, which facilitates the recovery of neutrophils, improving a patient’s ability to fight off infection. The release notes that patients who qualify for this treatment receive a single intravenous injection of stem cells from the modified cord blood. The cells are cultured with nicotinamide.

The approval follows clinical trial evaluations deeming Omisirge as safe and effective. The clinical trials conducted recruited 125 patients between 12 and 65. Each patient had undergone a stem cell transplant involving myeloablation through radiation or chemotherapy to treat blood cancer.

Researchers monitored each participant’s neutrophil levels and noted infection incidence. Roughly 87% of patients who received the experimental treatment had recovered neutrophil levels and a median recovery time within 12 days of treatment.

Comparatively, only 83% of participants in the control group receiving umbilical cord blood alone had recovered neutrophil levels. Although some patients in the control group reached the targeted neutrophil levels, it took them longer to achieve the desired outcome, with a median recovery time nearly twice as long as the experimental group, at 22 days.

Beyond neutrophil rates, the researchers concluded that the treatment Omisirge was superior to treatment with cord blood at preventing infection. Within 100 days of stem cell transplant, 60% of the control group participants had developed a bacterial or fungal infection. Meanwhile, only 39% of the Omisirge participants had an infection in the same time frame, a 21% reduction.

“Today’s approval is an important advance in cell therapy treatment in patients with blood cancers,” said Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, in the press 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.”

Before this regular FDA approval, the treatment had received Priority Review, Breakthrough Therapy, and Orphan drug designations.

Although the drug has been approved through multiple routes, the FDA warns against potential side effects, including graft versus host disease, infusion reactions, engraftment syndrome, infection, and graft failure. Providers must use their discretion to weigh the benefits of the treatment for protecting patients after a stem cell transplant and the risks associated with therapy.