The FDA approved abatacept to prevent acute graft versus host disease (aGVHD) for adults and children over the age of two undergoing bone marrow or stem cell transplantation from an unrelated donor, according to a press release from the FDA.
The FDA based its approval on two clinical studies that evaluated abatacept for safety and efficacy. The double-blind, placebo-controlled GVHD-1 trial included 186 stem cell transplant patients randomized to receive either abatacept or placebo combined with immunosuppressive drugs. Key endpoints for the trial included severe aGVHD-free survival and overall survival and moderate to severe acute aGVHD-free survival 6 months after transplantation.
Researchers observed a numerical but not statistically significant improvement in the rate of severe aGVHD survival among patients randomized to abatacept (87%) compared with those randomized to placebo (75%). The overall survival rate was 97% in the abatacept arm compared to 84% in the placebo arm. In addition, for patients with moderate to severe aGVHD, patients in the abatacept arm had a 50% survival rate compared to 32% in the placebo group.
The registry-based GVHD-2 clinical study used real-world data from the Center for International Blood and Marrow Transplant. The study evaluated 54 patients who received abatacept after stem cell transplantation from a mismatched unrelated donor compared to 162 patients given only immunosuppressive drugs. Patients in the abatacept arm had a 98% overall survival rate compared to 75% in the immunosuppressive group.
“Acute graft versus host disease can affect different parts of the body and become a serious post-transplant complication,” said Richard Pazdur, MD, director of the FDA’s Oncology Center for Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research, in the release. “By potentially preventing the disease, more patients may successfully undergo bone marrow or stem cell transplantation with fewer complications.”
The most common side effects for abatacept include anemia, hypertension, and cytomegalovirus reactivation/cytomegalovirus infection.
The FDA previously granted abatacept Breakthrough, Orphan Drug, and Priority Review designations to treat aGVHD. Abatacept is also approved to treat adults with rheumatoid arthritis and psoriatic arthritis and children with polyarticular idiopathic arthritis.
