Omisirge: A New Cell Therapy for Severe Aplastic Anemia and Blood Cancers

Omisirge (omidubicel-onlv) is a type of cell therapy made from umbilical cord blood. It helps your body make new blood cells after a stem cell transplant. What makes Omisirge different is that the cells are specially grown and preserved before being given to you, which may help them work better.

Omisirge is now approved by the FDA to treat a rare condition called severe aplastic anemia (SAA). SAA happens when your bone marrow stops making enough blood cells, which can lead to serious infections, bleeding, or fatigue. Many people with SAA don’t have a matched donor for a transplant, which limits their treatment options. Omisirge offers a new choice for people who haven’t responded to other treatments or don’t have a matched donor. Omisirge was first approved to treat certain blood cancers.

This approval is especially important because it’s the first time a cell therapy has been approved specifically for SAA. In studies, people treated with this medicine had a faster return to normal blood cell levels and fewer long-term complications like graft-versus-host disease (GVHD), a condition where the new cells attack your body.

Why Was It Approved?

Omisirge was approved because it helped people with severe aplastic anemia (SAA) recover their white blood cells more quickly and consistently after a transplant. In a study, most people had strong and lasting white blood cell recovery within about three months. Many also no longer needed transfusions for red blood cells or platelets.

The average time to white blood cell recovery was about 11 days, which is faster than expected. Importantly, no one in the study developed long-term GVHD. Only mild, short-term GVHD was seen in some people.

Serious side effects were reported, including infections, high blood sugar, low platelets, and breathing problems. One person died from a viral infection.

These results led the FDA to approve this medicine as a new treatment option for people with SAA who need a stem cell transplant.

What Do I Need to Know?

Omisirge is given as a one-time intravenous (IV) infusion through a vein. It comes in two parts that are given one after the other. Before getting this medicine, you’ll need a special treatment to prepare your body, called conditioning. The infusion must be done at a hospital with a transplant center that has experience with stem cell transplants.

Your health care provider will watch you closely for side effects during and after the infusion. These may include allergic reactions, infections, or problems with your new blood cells. If you’ve had allergic reactions to certain ingredients like DMSO or dextran 40, or to some antibiotics, be sure to tell your provider.

This medicine is not something you can pick up at a pharmacy. It’s made for specific people and must be matched to your identity.