FDA Approves Acute Graft Versus Host Disease Prophylaxis

Published on: 

The approval represents the first ever treatment for the disease in patients aged 2 years and older.

The US Food and Drug Administration (FDA) has approved abatacept (Orencia) as the first ever prophylaxis of acute graft versus host disease (aGVHD).

The approval allows clinicians to prescribe the drug for individuals 2 years and older undergoing hematopoietic stem cell transplantation from an unrelated donor.

The Disease

aGVHD occurs when donor bone marrow or stem cells attack the graft recipient in combination with certain immunosuppressants. The disease is a potentially fatal complication when the donor’s immune cells view the recipients body as foreign.

The approval is based on the incorporation of real world evidence as 1 of the components to determine clinical effectiveness, part of ongoing efforts for the FDA to incorporate the use of high-quality real world evidence to support regulatory decisions.

"Acute graft versus host disease can affect different parts of the body and become a serious post-transplant complication," said Richard Pazdur, M.D., director of the FDA's Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA's Center for Drug Evaluation and Research, in a statement. "By potentially preventing the disease, more patients may successfully undergo bone marrow or stem cell transplantation with fewer complications."

Clinical Trials

Abatacept approval is also based on 2 trials in patients aged 6 years and older who underwent stem cell transplantation from a matched or mismatched unrelated donor.

The first study was a double-blind, placebo-controlled trial involving 186 patients who underwent stem cell transplantation from a matched unrelated donor. Each patient randomly received either abatacept or a placebo in combination with immunosuppressive drugs.

Overall, severe aGVHD-survival was 87% in the abatacept group, compared to 75% in the placebo group, as well as a 97% overall survival rate compared to 84% in the placebo group.

The investigators also found moderate-severe aGVHD-free survival was 50% in the treatment group, compared to 32% in the placebo group.


For safety, the most common side effects were anemia, hypertension, cytomegalovirus (CMV) reactivation/CMV infection, fever, pneumonia, nosebleed, decreased levels of specific white blood cells called CD4 lymphocytes, increased levels of magnesium in the blood, and acute kidney injury.

Patients should also be monitored for Epstein-Barr virus reactivation.