FDA Accepts Biologics License Application for BMS aGvHD Therapy

August 26, 2021 - Bristol Myers Squibb recently announced that FDA accepted a supplemental biologics license application for its Acute Graft Versus Host Disease (aGvHD) therapy, Orencia. 

Orencia is intended to prevent moderate to severe aGvHD in patients six years of age and older receiving unrelated donor hematopoietic stem cell transplantation (HSCT). 

Stem cell transplants include infusion of donor T-cells. Generally, patients who receive stem cell transplants from unrelated and human leukocyte antigens (HLA)-mismatched donors are at high risk for developing aGvHD.

GvHD occurs when the donor T-cells recognize the patient’s healthy cells as foreign and start attacking healthy tissues and organs, a Bristol Myers Squibb spokesperson said. 

Leslie Kean, MD, PhD, director of the pediatric stem cell transplantation program at Boston Children’s Hospital/Dana-Farber Cancer Institute and lead study investigator, stated that there is a “tremendous need” to expand the stem cell donor pool by lowering the risk of aGvHD in both adults and children receiving unrelated donor stem cell transplants.

Between 30 and 70 percent of transplant recipients develop aGvHD, depending on donor type, transplant technique, and other features. 

Orencia binds to and inhibits protein targets involved in co-stimulation, inhibiting T-cell activation. 

Bristol Myers Squibb submitted a biologics license application to FDA based on Phase 2 ABA2 trial results and a registry trial based on real-world evidence. 

The ABA2 trial assessed the impact of Orencia to prevent severe aGvHD when added to a standard GvHD prophylactic regimen. Researchers enrolled patients with hematologic malignancies receiving a stem cell transplant from an unrelated, HLA-matched or mismatched donor.

Trial results from ABA2 showed that Orencia significantly reduced severe aGvHD and associated morbidity without an increase in disease relapse. The findings of the real-world analysis were consistent with those of ABA2.

Orencia is currently approved to treat various arthritic conditions.

In 2005, FDA approved Orencia to help reduce symptoms of moderate to severe rheumatoid arthritis in adults. And in 2017, the agency approved Orencia as an injection or intravenous infusion to treat PsA. Orencia may be taken alone or in combination with other PsA treatments. 

FDA granted the application priority review and assigned a prescription drug user fee act goal date for Orencia of December 2021. 

“For patients who receive unrelated donor stem cell transplants, in particular for racial and ethnic minority patient populations, there is a heightened risk of developing aGvHD, a potentially life-threatening medical complication for which there are no approved preventive therapies,” Mary Beth Harler, MD, head of immunology and fibrosis development at Bristol Myers Squibb, said in the recent announcement. 

“We look forward to working with the FDA to bring Orencia to this new patient population and employ pathbreaking science in an effort to address unmet needs of underserved patients,” Harler continued.