FDA Grants Breakthrough Therapy Designation to Abatacept for Acute GVHD Prevention

December 4, 2019

The FDA granted a breakthrough therapy designation to abatacept for the prevention of moderate to severe acute graft-versus-host disease in hematopoietic stem cell transplants from unrelated donors.

The FDA granted a breakthrough therapy designation to abatacept (Orencia) for the prevention of moderate to severe acute graft-versus-host disease (GvHD) in hematopoietic stem cell transplants from unrelated donors, according to Bristol-Myers Squibb.

“While ideally we prefer using fully matched transplants from a sibling for the treatment of hematologic cancers, only the minority of patients have such a sibling,” lead study investigator Leslie Kean, MD, director of the Stem Cell Transplantation Program, Dana Farber/Boston Children’s Cancer and Blood Disorders Center, said in a press release. “A therapy that lowers the risk of GvHD in unrelated stem cell transplants would potentially allow more patients to receive a transplant, which typically is the last option to treat hematologic cancers after other therapies have been used unsuccessfully.”

The agency’s designation was based on findings from an investigator-initiated phase II study, designed to evaluate abatacept on the prevention of severe acute GvHD, when added to a standard GvHD prophylactic regimen administered to patients with hematologic malignancies receiving a stem cell transplant from an unrelated, HLA-matched or mismatched donor.

Abatacept is currently approved to treat various arthritic conditions. The immunomodulator binds to and inhibits protein targets involved in co-stimulation, which inhibits T-cell activation.

“We are excited about the potential of (abatacept) to improve outcomes for patients receiving unrelated stem cell transplants. We believe the data could lead to an expansion of the donor pool for stem cell transplants in some patient populations where fully matched unrelated donor transplants have rarely been available,” Brian Gavin, PhD, development lead of the drug at Bristol-Myers Squibb, said in the release. “We look forward to working with the FDA and making ORENCIA the first approved therapy for the prevention of acute GvHD.”

The FDA’s breakthrough therapy designation program is intended to expedite the development and review of medicines for serious or life-threatening diseases with preliminary clinical evidence that the investigational therapy may offer substantial improvement on at least 1 clinically significant endpoint over available therapy.

Reference:
Bristol-Myers Squibb. Bristol-Myers Squibb Announces U.S. FDA Breakthrough Therapy Designation for ORENCIA® (abatacept) to Help Prevent Acute Graft-Versus-Host Disease, a Potentially Life-Threatening Complication After Stem Cell Transplant. Published December 4, 2019. https://news.bms.com/press-release/rd-news/bristol-myers-squibb-announces-us-fda-breakthrough-therapy-designation-orencia. Accessed December 4, 2019.