The FDA has set a PDUFA date of December 23, 2021.
The FDA has accepted Bristol-Myers Squibb’s supplemental Biologics License Application (sBLA) for Orencia (abatacept) for the prevention of moderate-to-severe acute graft versus host disease (aGvHD) in patients 6 years of age and older receiving unrelated donor hematopoietic stem cell transplantation. The FDA granted the application priority review and assigned a PDUFA goal date of December 23, 2021.
If approved, Orencia would become the first therapy for the prevention of aGvHD.
“While stem cell transplants are an effective treatment for aggressive leukemias and other hematological malignancies, patients who receive stem cell transplants from unrelated and human leukocyte antigens (HLA)-mismatched donors are at high risk for developing aGvHD,” lead investigator Leslie Kean, M.D., Ph.D., director of the pediatric stem cell transplantation program at Boston Children’s Hospital/Dana-Farber Cancer Institute, said in a statement.
The sBLA submitted to the FDA is based on results from the phase 2 ABA2 trial and a registry trial based on real-world evidence. The ABA2 trial assessed the impact of Orencia on the prevention of severe aGvHD, when added to a standard GvHD prophylactic regimen administered to patients with hematologic malignancies receiving a stem cell transplant from an unrelated, HLA-matched or mismatched donor. A mismatch in HLA increases the risk of GvHD.
Results from ABA2 showed that treatment with Orencia resulted in a reduction in severe aGvHD. Investigators found from immunologic analysis that Orencia controlled T-cell activation in treated patients. Investigators also found that the decreased in aGvHD was not accompanied by an increase in the risk of relapse or infectious complications.
The findings of the real-world analysis were consistent with those of ABA2, according to executives from Bristol Myers Squibb.
Between 30% and 70% of transplant recipients develop aGvHD. Orencia binds to and inhibits protein targets involved in co-stimulation.
Orencia is approved to treat adults with rheumatoid arthritis and psoriatic arthritis, as well as children with polyarticular juvenile idiopathic arthritis.