FDA approves Orencia for prevention of acute GVHD
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The FDA approved abatacept in combination with a calcineurin inhibitor and methotrexate for prevention of acute graft-versus-host disease.
Abatacept (Orencia, Bristol Myers Squibb) is an immunomodulator designed to disrupt the continuous cycle of T-cell activation. It is approved in the United States for treatment of certain patients with arthritis.
The approval for GVHD prevention applies to adults and children aged 2 years or older undergoing hematopoietic stem cell transplantation from an unrelated donor.
“Acute [GVHD] can affect different parts of the body and become a serious post-transplant complication,” Richard Pazdur, MD, director of 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 an agency-issued press release. “By potentially preventing the disease, more patients may successfully undergo bone marrow or stem cell transplantation with fewer complications.”
The approval of abatacept — the first for a drug to prevent acute GVHD — incorporated real-world evidence as a component of the clinical efficacy determination, according to the FDA.
Two separate studies evaluated the safety and efficacy of abatacept plus immunosuppressant therapy for patients aged 6 years and older who received stem cell transplants from a matched or mismatched unrelated donor.
The first study, GVHD-1, included 186 patients who underwent transplantation from a matched unrelated donor. Researchers randomly assigned patients to abatacept or placebo plus immunosuppressive drugs. Although abatacept did not appear to significantly improve acute (grade III to grade IV) GVHD-free survival rates compared with placebo (87% vs. 75%), patients who received the drug demonstrated higher rates of OS (97% vs. 84%) and moderate-to-severe (grade II to grade IV) acute GVHD-free survival (50% vs. 32%).
The second study, GVHD-2, used real-world data from the Center for International Blood and Marrow Transplant Research to analyze outcomes of 54 patients who received abatacept prophylaxis plus standard immunosuppressive drugs compared with those of 162 patients who received standard immunosuppressive drugs alone. Results showed higher OS at 6 months after transplantation among those who received abatacept (98% vs. 75%).
The most common adverse events associated with abatacept for acute GVHD include anemia, hypertension, cytomegalovirus (CMV) reactivation/CMV infection, fever, pneumonia, nosebleed, decreased levels of CD4 lymphocytes, increased levels of magnesium in the blood and acute kidney injury. Patients should be monitored for reactivation of Epstein-Barr virus and receive preventive medication for Epstein-Barr virus infection prior to treatment initiation and for 6 months after transplantation, according to the press release. Patients also should be monitored for CMV infection/reactivation for 6 months after transplant.
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