The US Food and Drug Administration (FDA) has approved Gamida Cell’s Omisirge (omidubicel-onlv) cell therapy to reduce the risk of infection following stem cell transplantation in blood cancer patients.
Omisirge is specifically indicated for individuals aged 12 years and older who are planned for umbilical cord blood transplantation following a myeloablative conditioning regimen, such as radiation or chemotherapy.
Stem cell transplantation is a common treatment for blood cancers, which represent about 10% of annual US cancer cases. One source of healthy stem cells is umbilical cord blood.
Before receiving this kind of transplant, patients will generally undergo a course of treatments to remove their own stem cells and prepare the body for new ones, which may weaken the immune system and increase the risk of severe infections.
Omisirge, administered as a single intravenous dose, is composed of human allogeneic stem cells from umbilical cord blood that are processed and cultured with nicotinamide – a form of vitamin B3. Each dose is patient-specific, containing healthy stem cells from a pre-screened donor.
The FDA’s decision was supported by results from a study of 125 blood cancer patients comparing transplantation of Omisirge to transplantation of umbilical cord blood. Efficacy was based on the amount of time needed for recovery of the patients’ neutrophils – a type of white blood cell that helps protect the body from infections – and the incidence of infections following transplantation.
In the Omisirge cohort, 87% of patients achieved neutrophil recovery with a median of 12 days following treatment, compared to 83% of patients who received umbilical cord blood transplantation and who achieved neutrophil recovery with a median of 22 days.
Bacterial or fungal infections by 100 days following transplantation were seen in 39% of patients receiving Omisirge versus 60% of those receiving umbilical cord blood.
Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said the approval was “an important advance” in cell therapy treatment for blood cancer patients. “Hastening the return of the body’s white blood cells can reduce the possibility of serious or overwhelming infection associated with stem cell transplantation.”
Abbey Jenkins, president and chief executive officer of Gamida, said: “The FDA approval of Omisirge is a major advancement in the treatment of patients with haematologic malignancies that we believe may increase access to stem cell transplant and help improve patient outcomes.”