FDA Greenlights BMS’s Orencia for Acute GVHD – Partly on RWE

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Bristol Myers Squibb’s rheumatoid arthritis drug Orencia won approval for the prevention of acute graft versus host disease (aGvHD) in patients two years of age and older who have received unrelated donor hematopoietic stem cell transplantation. It is the first such drug approved by the U.S. Food and Drug Administration for this indication.

Orencia snagged approval from the FDA under Priority Review. In its announcement, the FDA said Orencia, an immunomodulator that disrupts the continuous cycle of T-cell activation, is the first drug approved as a prophylaxis for aGVHD prevention that included real-world evidence as one component of clinical efficacy. Acute GVHD is a potentially fatal complication that can occur after stem cell transplantation. This happens when the donor's immune cells view the recipient's body as foreign and then attack the body. The chances of developing aGVHD increase when the donor and recipient are not related or are not a perfect match.

The latest approval for Orencia was generated in two different studies, including the Phase II ABA2 trial, which assessed the impact of Orencia on prevention of severe aGvHD. This was done when Orencia was added to a standard GvHD prophylactic regimen administered to patients with hematologic malignancies who received a stem cell transplant from an unrelated, HLA-matched or mismatched donor. Results showed that when Orencia was added, there was a significant reduction in severe aGvHD and associated morbidity without an increase in disease relapse. Patients who received Orencia saw a 98% overall survival rate compared to 75% for patients who received standard immunosuppression alone.

In additional data provided by the GVHD-1 study, patients with severe disease who received Orencia saw a 97% overall survival rate compared to 84% for placebo. For moderate-severe aGVHD-free survival, patients who received Orencia saw a 50% rate compared to 32% for patients who received a placebo.

Dr. Richard Pazdur, M.D., 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 acute graft versus host disease can be a serious post-transplant complication in patients.

“By potentially preventing the disease, more patients may successfully undergo bone marrow or stem cell transplantation with fewer complications,” Prazdur said in a statement.

Orencia won Breakthrough Therapy Designation, as well as Orphan Drug Designation for this indication.

The Priority Review for Orcenica was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence that offers a framework for concurrent submission and review of oncology drugs among international partners. 

In addition to rheumatoid arthritis, Orencia has also been approved for the treatment of polyarticular juvenile idiopathic arthritis and adult psoriatic arthritis.  

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