FDA News

FDA grants Orencia breakthrough therapy designation to prevent acute GVHD

The FDA granted breakthrough therapy designation to abatacept for prevention of moderate to severe acute graft-versus-host disease among individuals who undergo hematopoietic stem cell transplantation from unrelated donors.

There are no approved therapies for prevention of acute GVHD, a potentially life-threatening complication that affects up to 40% of patients who undergo stem cell transplants from unrelated donors.

Abatacept (Orencia, Bristol-Myers Squibb) is an immunomodulator that binds to and inhibits protein targets involved in co-stimulation, thereby inhibiting T-cell activation.

The FDA based the breakthrough therapy designation on results from a phase 2 trial that evaluated the addition of abatacept to a standard GVHD prophylactic regimen for patients with hematologic malignancies who received a stem cell transplant from an unrelated HLA-matched or -mismatched donor.

“[Although] 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 at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, said in a Bristol-Myers Squibb-issued press release. “A therapy that lowers the risk [for] 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.”

Abatacept is approved in the United States for treatment of specific types of arthritic conditions among children and adults.

“We are excited about the potential of Orencia to improve outcomes for patients receiving unrelated stem cell transplants,” Brian Gavin, PhD, development lead for Orencia at Bristol-Myers Squibb, said in the release. “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. We look forward to working with the FDA and making Orencia the first approved therapy for the prevention of acute GVHD.”

The FDA granted breakthrough therapy designation to abatacept for prevention of moderate to severe acute graft-versus-host disease among individuals who undergo hematopoietic stem cell transplantation from unrelated donors.

There are no approved therapies for prevention of acute GVHD, a potentially life-threatening complication that affects up to 40% of patients who undergo stem cell transplants from unrelated donors.

Abatacept (Orencia, Bristol-Myers Squibb) is an immunomodulator that binds to and inhibits protein targets involved in co-stimulation, thereby inhibiting T-cell activation.

The FDA based the breakthrough therapy designation on results from a phase 2 trial that evaluated the addition of abatacept to a standard GVHD prophylactic regimen for patients with hematologic malignancies who received a stem cell transplant from an unrelated HLA-matched or -mismatched donor.

“[Although] 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 at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, said in a Bristol-Myers Squibb-issued press release. “A therapy that lowers the risk [for] 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.”

Abatacept is approved in the United States for treatment of specific types of arthritic conditions among children and adults.

“We are excited about the potential of Orencia to improve outcomes for patients receiving unrelated stem cell transplants,” Brian Gavin, PhD, development lead for Orencia at Bristol-Myers Squibb, said in the release. “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. We look forward to working with the FDA and making Orencia the first approved therapy for the prevention of acute GVHD.”