FDA Oks Bristol Myers' Orencia To Prevent Graft Versus Host Disease

(RTTNews) - Bristol Myers Squibb's Orencia has received approval from the U.S. Food and Drug Administration for prophylaxis or prevention of acute graft versus host disease or aGVHD.

This is the first FDA drug approval for prevention of aGVHD, which is a condition that occurs when donor bone marrow or stem cells attack the graft recipient, in combination with certain immunosuppressants. It incorporates real world evidence or RWE as one component of the determination of clinical effectiveness.

Acute GVHD is a potentially fatal complication that can occur after stem cell transplantation when the donor's immune cells (the graft) view the recipient's body (the host) as foreign, and the donated cells attack the body.

According to the agency, the chances of developing aGVHD increase when the donor and recipient are not related or are not a perfect match.

The newly approved Orencia may 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.

The company evaluated the safety and efficacy of 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 in two separate studies.

The first study, GVHD-1, was a double-blind, placebo-controlled trial of 186 patients. While severe aGVHD-survival was not significantly improved in patients who received Orencia compared to patients who received a placebo, patients who received Orencia saw a 97 percent overall survival rate compared to 84 percent for patients who received a placebo.

Additional evidence of effectiveness was provided by GVHD-2, a registry-based clinical study conducted using real world data from the Center for International Blood and Marrow Transplant Research in patients. In this, patients who received Orencia saw a 98 percent overall survival rate compared to 75 percent for patients who received standard immunosuppression alone.

Orencia received Breakthrough, Orphan Drug and Priority Review designations for this indication.

The company develops the product with partial support from the FDA's Orphan Products Grants Program, which provides grants for clinical studies on safety and efficacy of products for use in rare diseases or conditions.

The FDA said, "Acute graft versus host disease can affect different parts of the body and become a serious post-transplant complication. By potentially preventing the disease, more patients may successfully undergo bone marrow or stem cell transplantation with fewer complications."

Orencia was originally approved by the FDA in 2005 for the treatment of adult rheumatoid arthritis. The drug is also approved for the treatment of polyarticular juvenile idiopathic arthritis and adult psoriatic arthritis.