The FDA Grants Breakthrough Therapy Designation for ORENCIA as a Treatment for Graft vs Host Disease

According to a story from Business Wire, the biopharmaceutical company Bristol-Myers Squibb has recently announced that the US Food and Drug Administration (FDA) has granted the company’s drug abatacept (marketed as ORENCIA) Breakthrough Therapy designation for acute graft-versus-host disease, a rare and potentially lethal complication of stem cell transplants. At this juncture, there are no approved therapies for severe acute graft-versus-host disease.

About Graft-versus-Host Disease (GvHD)

Graft-versus-Host disease is a potentially fatal medical complication that can appear in a patient that has recently received a transplant operation from another person. It is most commonly triggered by bone marrow transplants but it can also occur with solid organ transplants as well. The disease occurs when white blood cells that remain in the transplanted tissue begin to identify the host body as a foreign intruder and begin to attack the host’s cells. Many recipients of transplanted tissue have weakened immune systems which means that their own body is often incapable of preventing the attack from beginning. Symptoms of graft-versus-host disease include skins rashes, gastrointestinal and liver damage, and damage to the mucosa. Long term disease may see the attack spread to other areas as well, such as the host immune system, the exocrine glands, lungs, and connective tissue. Treatment often includes immunosuppressants such as steroids and certain chemotherapy agents. To learn more about graft-versus-host disease, click here.

About Breakthrough Therapy Designation

Breakthrough Therapy designation is a special FDA program that is used to speed up the development of therapies intended to treat serious, life threatening diseases. This designation is typically allotted to a therapy that has displayed potential advantages over current therapies in early clinical testing. A drug that earns this designation is eligible for more extensive organizational commitment and guidance on drug development from the agency and all benefits from Fast Track designation (Rolling Review, Priority Review, Accelerated Approval, etc.).


ORENCIA is an immunomodulator drug that is currently approved for several uncommon forms of arthritis, such as juvenile idiopathic arthritis, psoriatic arthritis, and rheumatoid arthritis. Graft-versus-host disease is a particular danger during transplant operations between unrelated donors. If ORENCIA is ultimately approved for this indication, patients facing this dangerous complication would have a greater chance of a favorable treatment outcome.

Share this post