The U.S. Food and Drug Administration (FDA) has approved omidubicel (Omisirge), an allogeneic cord blood-based cell therapy to quicken the recovery of neutrophils and reduce the risk of infection.
The product is approved for patients 12 years and older with blood cancers planned for umbilical cord blood transplantation following radiation or chemotherapy.
Each type of blood cancer is a rare cancer but collectively, blood cancers (e.g., lymphomas, leukemias, multiple myeloma) represent about 10% of all cases of cancer each year in the U.S.
Stem cell transplantation is a common therapy for rare blood cancers but radiation or chemotherapy that precedes the stem cell transplantation can weaken the person’s immune system and make them vulnerable to severe, and occasionally, deadly infections until the stem cells develop into white blood cells (e.g., neutrophils). Omidubicel, 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) that can speed up the development of neutrophils.
The approval of omidubicel was largely based on a randomized, multicenter study comparing transplantation of omidubicel to transplantation of umbilical cord blood, in subjects between the ages of 12 and 65 years. The study enrolled a total of 125 individuals with blood cancers. In the study, 87% of patients given omidubicel achieved neutrophil recovery with a median of 12 days compared to 83% of patients in the control group who achieved neutrophil recovery with a median of 22 days.
Bacterial or fungal infections were seen in 39% of patients receiving omidubicel compared to 60% of patients in the control group.
The most common adverse reactions associated with omidubicel included infections, GvHD, and infusion reactions.
Omidubicel comes with a Boxed Warning for infusion reactions, graft versus host disease (GvHD), engraftment syndrome, and graft failure.
To stay up-to-date on the latest FDA approvals, go to checkrare.com/2023-orphan-drugs-pdufa-dates-and-fda-approvals/
