The FDA has given the green light to Omisirge (omidubicel-onlv), an allogeneic cell therapy derived from modified cord blood, which aims to accelerate the recovery of neutrophils and lower the risk of infection in patients with blood cancers. The treatment is designed for adults and children aged 12 and above who are scheduled for umbilical cord blood transplantation after undergoing myeloablative conditioning.
Blood cancers are a result of uncontrolled cell growth in the blood, which disrupts the normal function of blood cells. The abnormal growth usually starts in the bone marrow, where stem cells form into different types of blood cells with specific functions in the body. In the U.S., blood cancers account for approximately 10% of all cancer cases each year. Blood cancers can be life-threatening, with varying survival rates based on factors such as the type of blood cancer. In addition to being fatal, blood cancers can also cause severe and damaging effects on the body, resulting in symptoms such as fatigue, night sweats, bone and joint pain, weakness, weight loss, fever, and infections.
Stem cell transplantation is a common treatment for blood cancers that involves introducing healthy stem cells into the body to help restore the normal production and function of blood cells. Umbilical cord blood is a source of healthy stem cells. Before receiving this type of transplant, the patient typically undergoes a course of treatments to remove their own stem cells and prepare the body for the new stem cells. This process often includes radiation or chemotherapy, both of which can weaken an individual’s immune system. Consequently, the occurrence of severe and sometimes fatal infections is a common and serious risk associated with this treatment.
Omisirge is a one-time intravenous infusion composed of human allogeneic stem cells derived from umbilical cord blood that has been modified with nicotinamide, a form of vitamin B3. The therapy is unique to each patient and contains healthy stem cells obtained from a pre-screened allogeneic donor, meaning that it comes from a different individual rather than using the patient’s own cells.
A randomized, multicenter study was conducted to evaluate the safety and effectiveness of Omisirge in patients with confirmed blood cancers, between the ages of 12 and 65 years. The study enrolled a total of 125 subjects and compared the transplantation of Omisirge with the transplantation of umbilical cord blood. The efficacy of Omisirge was determined by measuring the time needed for recovery of neutrophils, which are a type of white blood cell that helps the body fight infections. Additionally, the incidence of infections following transplantation was also used to assess the effectiveness of Omisirge.
In a randomized, multicenter study involving 125 subjects between the ages of 12 and 65 with confirmed blood cancers, the safety and efficacy of Omisirge were compared to the transplantation of umbilical cord blood. The study found that 87% of subjects who received Omisirge achieved neutrophil recovery in a median time of 12 days, while 83% of subjects who received umbilical cord blood transplantation achieved neutrophil recovery in a median time of 22 days. Additionally, 39% of subjects who received Omisirge experienced bacterial or fungal infections within 100 days following transplantation, compared to 60% of subjects in the control group who received umbilical cord blood.
Before using Omisirge, potentially severe side effects should be taken into account and balanced against its benefits. The product’s label includes a Boxed Warning for infusion reactions, engraftment syndrome (a noninfectious fever and rash), graft failure (when new cells fail to produce blood cells), and graft-versus-host disease (GvHD), a condition where donor cells attack the recipient’s cells. These potential risks are similar to those associated with all approved umbilical cord products.
Omisirge treatment can lead to several adverse reactions, including infections, graft versus host disease, and infusion reactions. Therefore, patients receiving Omisirge should be monitored for signs and symptoms of these conditions. They should also be monitored for engraftment syndrome, graft failure, the transmission of serious infections or rare genetic diseases from the donor cells, and the development of secondary malignancies, which are cancers that can spread from the original site or emerge following treatment. Monitoring should continue throughout the patient’s life.
Source: FDA
