Patients receiving bone marrow or stem cell transplants face the prospect of a dangerous and potentially fatal immune response.This FDA approved Bristol Myers Squibb uses Orencia drugs as a way to avoid this condition, which is called acute graft-versus-host disease. Regulatory decisions make the drug the first drug approved to prevent complications of this transplant.
Acute graft-versus-host disease occurs when the immune cells of the donor cell treat the transplant recipient’s body as a foreign body. Therefore, the donated cells attack the body. When the donor and recipient are not related, or the donor cells are not perfectly matched, the risk of this complication is greater. The US Food and Drug Administration’s decision on Wednesday covers the use of Orencia in combination with immunosuppressive agents for adults and children 2 years of age or older who receive bone marrow or stem cell transplants from unrelated donors.
Orencia is a fusion protein designed to bind two targets and block T cell activation. BMS drugs are already on the market. It was first approved for the treatment of adult human rheumatoid arthritis in 2005. The drug has continued to receive additional approvals for adult psoriatic arthritis and juvenile idiopathic arthritis. According to BMS financial data, in 2020, sales of the drug exceeded US$3.1 billion, an increase of 6% over the previous year.
When graft-versus-host disease has appeared, the drugs used to treat it include steroids and immunosuppressive drugs. In July, the FDA approves Rezurock, A Kadmon Holdings drug developed to treat chronic graft-versus-host disease.The approval triggered Sanofi acquires the companyPrior to Orencia’s new approval, there was no standard drug regimen for the prevention of acute graft-versus-host disease. According to the Leukemia and Lymphoma Association, Different medical institutions provide different drug combinations.
Orencia’s new approval is based on the results of two clinical trials. The first is a placebo-controlled study that recruited 186 patients who received stem cell transplants from matched unrelated donors. The study assessed the severity of acute graft-versus-host disease and the survival rate of patients. The FDA stated that in the Orencia group, the survival rate of patients with severe acute graft-versus-host disease did not increase significantly—87%, compared with 75% in the placebo group. However, the agency noted that the survival rate for patients receiving the study drug was 97%, compared with 84% for the placebo group. For moderate to severe cases of acute graft-versus-host disease, the survival rate in the Orencia group was 50%, compared to 32% in the placebo group.
The second clinical study evaluated real evidence from data provided by the International Center for Blood and Bone Marrow Transplant Research. These data come from patients who received stem cell transplants from unmatched unrelated donors. A total of 54 patients who received BMS drugs combined with immunosuppressive drugs were compared with 162 patients who received only immunosuppressive drugs. The FDA stated that after six months, the survival rate of patients receiving Orencia treatment was 98%, while the survival rate of patients receiving standard immunosuppressive therapy was 75%.
Side effects reported in clinical trials include anemia, hypertension, reactivation of cytomegalovirus (CMV) and infection of the virus, fever, and pneumonia. The FDA stated that patients receiving BMS drugs should be monitored for reactivation of Epstein-Barr virus, and they should also receive preventive drug treatment against the virus before starting treatment and within six months after transplantation. The FDA stated that clinicians should also monitor patients for CMV reactivation and infection within 6 months after transplantation.
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