FDA Approves Orencia for GVHD Prevention After Hematopoietic Stem Cell Transplant

The Food and Drug Administration (FDA) approved Orencia (abatacept) plus other immunosuppressive drugs for the prevention of acute graft-versus-host disease (aGVHD) in adult and pediatric patients aged 2 years and older who underwent a hematopoietic stem cell transplant from an unrelated donor.

This is the first FDA approval for the prevention of aGVHD, a dangerous condition where the recipient’s body sees the donor immune cells as foreign and, in turn, attacks them.

"Acute graft versus host disease can affect different parts of the body and become a serious post-transplant complication," said Dr. Richard Pazdur, 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.

The approval is based off findings from two studies. The first, named GVHD-1, was a placebo-controlled trial involving 186 patients who underwent stem cell transplantation and were randomized to receive either Orencia plus immunosuppressive drugs or placebo plus immunosuppressive drugs. Though severe (grade 3/4) aGVHD-survival was not significantly improved for those receiving Orencia (87%) versus those receiving placebo (75%), the Orencia group did achieve a 97% overall survival rate, compared with 84% for the placebo group. When it came to moderate to severe aGVHD-survival, the Orencia group was significantly better at 50%, compared with 32% in patients who received the placebo.

The GVHD-2 study looked at real-world data from the Center for International Blood and Marrow Transplant Research. Data from 54 patients who had a stem cell transplant from a mismatched, unrelated donor and were given Orencia plus immunosuppressive drugs were analyzed. These data were compared with outcomes of 162 patients who were treated just with standard immunosuppressive drugs.

The main goal of GVHD-2 was to analyze overall survival six months after transplantation. Patients on Orencia fared far better – with a 98% overall survival rate compared with 75% for those on standard immunosuppressive drugs.

The most common side effects of Orencia, which is commonly used to treat rheumatoid arthritis, are anemia, high blood pressure, cytomegalovirus (CMV) reactivation or infection, fever, pneumonia, nosebleed, decrease of CD4 lymphocytes (a type of white blood cell), increase in magnesium levels in the blood and acute kidney injury. Patients on Orencia should be monitored for Epstein-Barr virus as well as CMV infection or reactivation.

Ongoing research continues to examine Orencia in GVHD prevention.

"By potentially preventing the disease, more patients may successfully undergo bone marrow or stem cell transplantation with fewer complications,” Pazdur said.

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