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

PRINCETON, NJ, USA I December 04, 2019 IBristol-Myers Squibb Company (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for ORENCIA® (abatacept) for the prevention of moderate to severe acute graft-versus-host disease (GvHD) in hematopoietic stem cell transplants from unrelated donors. There are no approved therapies for the prevention of acute GvHD, a potentially life-threatening medical complication that can impact patients receiving such transplants for the treatment of certain genetic diseases and hematologic cancers.1

Stem cell transplants have been shown to be an effective treatment for aggressive leukemias and other hematological malignancies, often representing the only therapeutic option for cure. However, some of their benefit is offset by the occurrence of severe acute GvHD, which impacts up to 40 percent of patients receiving stem cell transplants from unrelated donors with a mismatch in genes called human leukocyte antigens (HLA).2 These transplants are associated with a high rate of transplant-related mortality stemming largely from severe acute GvHD.3,4,5

“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,” said study lead investigator Leslie Kean, M.D., Director of the Stem Cell Transplantation Program, Dana Farber/Boston Children’s Cancer and Blood Disorders Center. “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.”

Stem cell transplant infusions include donor T-cells, a type of white blood cell that recognizes and destroys foreign invaders in the recipient’s body including cancer cells. GvHD occurs when the donor T-cells also recognize the patient’s healthy cells as foreign and start attacking healthy tissues and organs.1 T-cell activation requires a signaling process called co-stimulation. ORENCIA, a therapy currently approved to treat various arthritic conditions, binds to and inhibits protein targets involved in co-stimulation, thus inhibiting T-cell activation.

The Breakthrough Therapy Designation is based on findings from an investigator-initiated study supported by Bristol-Myers Squibb. This Phase 2 trial assessed the impact of ORENCIA 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. A mismatch in HLA increases the risk of GvHD.

Breakthrough Therapy Designation is an FDA program 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 one clinically significant endpoint over available therapy.

“We are excited about the potential of ORENCIA 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,” said Brian Gavin, Ph.D., development lead, ORENCIA, Bristol-Myers Squibb. “We look forward to working with the FDA and making ORENCIA the first approved therapy for the prevention of acute GvHD.”

About Acute Graft-versus-Host Disease

GvHD after a hematopoietic stem cell transplant occurs when transplanted donor T-cells recognize antigenic differences between the donor and the recipient and attack the recipient’s healthy tissue and organs.1 Acute GvHD impacts up to 40 percent of patients who receive stem cell transplants from unrelated and HLA-mismatched donors.2 This activation of T-cells can result in severe immune-mediated tissue damage to the host, with the skin, liver and gastrointestinal tract being the most common targets. Acute GvHD-mediated damage to these vital organs has been associated with increased morbidity and death.1


ORENCIA® is an immunomodulator that disrupts the continuous cycle of T-cell activation that characterizes Rheumatoid Arthritis.

U.S. Indications/Usage and Important Safety Information for ORENCIA® (abatacept)

Indication and Usage

Adult Rheumatoid Arthritis (RA): ORENCIA® (abatacept) is indicated for reducing signs and symptoms, inducing major clinical response, inhibiting the progression of structural damage, and improving physical function in adult patients with moderately to severely active RA. ORENCIA may be used as monotherapy or concomitantly with disease-modifying, anti-rheumatic drugs (DMARDs) other than tumor necrosis factor (TNF) antagonists.

Juvenile Idiopathic Arthritis (JIA): ORENCIA® (abatacept) is indicated for reducing signs and symptoms in patients 2 years of age and older with moderately to severely active polyarticular JIA. ORENCIA may be used as monotherapy or concomitantly with methotrexate (MTX).

Adult Psoriatic Arthritis (PsA): ORENCIA® (abatacept) is indicated for the treatment of adult patients with active PsA.

Important Limitations of Use: ORENCIA should not be administered concomitantly with TNF antagonists, and is not recommended for use concomitantly with other biologic RA therapy, such as anakinra.

About Bristol-Myers Squibb Immunology

With a robust pipeline of immunomodulatory therapies, Bristol-Myers Squibb is committed to the discovery and development of transformational medicines that could lead to long-term remission in patients with immune-mediated diseases. As we discover more about the immune system in such diseases with substantial unmet medical needs, the potential for developing novel therapies and biomarkers of response that target specific pathways in the immune system continues to drive our research efforts.

About Bristol-Myers Squibb

Bristol-Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol-Myers Squibb, visit us at or follow us on LinkedIn , Twitter , YouTube, Facebook and Instagram.


  1. Leukemia & Lymphoma Society. Graft-Versus Host Disease Fact Sheet No. 32. Updated 12/06/2017. Accessed November 7, 2019.
  2. Jacobsohn DA, Vogelsang GB. Acute graft versus host disease. Orphanet J. Rare Dis. 2007;2:35
  3. Bunin N, Carston M, Wall D, et al. Unrelated marrow transplantation for children with acute lymphoblastic leukemia in second remission. Blood 2002;99:3151-3157.
  4. Woolfrey A, Klein JP, Haagenson M, et al. HLA-C Antigen mismatches are associated with worse outcomes in unrelated donor peripheral blood stem cell transplantation. Biol Blood Marrow Transplant 2011.
  5. Jagasia M, Arora M, Flowers ME, et al. Risk factors for acute GVHD and survival after hematopoietic cell transplantation. Blood 2012;119:296-307.

  SOURCE: Bristol-Myers Squibb

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