The FDA today approved Bristol Myers Squibb’s Orencia (abatacept) for the prophylaxis (prevention) of acute graft versus host disease (aGVHD).
According to an FDA release, this is the first FDA drug approval for aGVHD prevention, incorporating real-world evidence as a component of the determination of clinical effectiveness.
Orencia — originally approved by the FDA in 2005 for treating adult rheumatoid arthritis and also approved for treating polyarticular juvenile idiopathic arthritis and adult psoriatic arthritis — may now be used in adults and pediatric patients two years of age or older undergoing hematopoietic stem cell transplantation (commonly known as bone marrow transplantation or stem cell transplantation) from an unrelated donor.
Bristol Myers Squibb had previously received breakthrough, orphan drug and priority review designations with the development of the product partially supported by the FDA’s Orphan Products Grants Program.
Two separate studies evaluated the safety and efficacy Orencia in combination with immunosuppressant therapy in patients age six years and older who underwent stem cell transplantation from a matched or mismatched unrelated donor. The most common side effects included 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 who receive Orencia should be monitored for Epstein-Barr virus reactivation in accordance with institutional practices and receive preventative medication for Epstein-Barr virus infection before starting treatment and for six months post-transplantation. Patients should also be monitored for CMV infection/reactivation for six months post-transplant.
“Acute graft versus host disease can affect different parts of the body and become a serious post-transplant complication,” Dr. Richard 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, said in the release. “By potentially preventing the disease, more patients may successfully undergo bone marrow or stem cell transplantation with fewer complications.”