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FDA approves first drug to prevent graft-versus-host disease

Bristol Myers Squibb’s arthritis drug, Orencia, can now be given to adults and children undergoing stem cell transplants to prevent the life-threatening condition


Bone marrow transplants for the treatment of leukaemia and other blood cancers have a long history, with the first procedure in 1957 in identical twins leading its pioneer, Dr E Donnall Thomas, to win a Nobel Prize.

Huge strides have been made since then and haematopoietic stem cell transplantation (HSCT) is now an effective treatment –even a cure– for haematological malignancies and other non-malignant haematological diseases.

Yet, up to 70% of patients that receive stem cell transplants, especially those who receive them from an unrelated donor, are impacted by acute graft-versus-host disease (aGvHD), where the transplanted donor T-cells attack the recipient’s healthy tissue and organs.

Now, health professionals have a new weapon in their arsenal after the US Food and Drug Administration (FDA) has approved the first therapy designed to prevent aGvHD – Bristol Myers Squibb’s (BMS) arthritis drug, Orencia (abatacept).

Orencia can now be given, in combination with a calcineurin inhibitor and methotrexate, to adults and children aged two years and older who are undergoing haematopoietic stem cell transplantation from a matched or 1 allele-mismatched unrelated donor.

“Acute graft-versus-host disease can affect different parts of the body and become a serious post-transplant complication,” said Dr Richard Pazdur, director of the FDA’s Oncology Center of Excellence. “By potentially preventing the disease, more patients may successfully undergo bone marrow or stem cell transplantation with fewer complications.”

The approval is based on data from two trials. In the phase 2 GVHD-1 study, while patients who received Orencia saw a 97% overall survival rate compared to 84% for patients who received a placebo. The second trial, a clinical study known as GVHD-2, used real-world data collected by the Center for International Blood and Marrow Transplant Research (CIBMTR) which showed that patients who received Orencia saw a 98% overall survival rate compared to 75% for patients who received standard immunosuppression alone.

Tina Deignan, senior vice president at BMS, welcomed the approval, highlighting the comparatively higher risk to racial and ethnic minority populations in the US due to difficulty finding appropriately matched donors. “With this fourth indication for Orencia, Bristol Myers Squibb draws on its legacy and expertise in both immunology and haematology to deliver an important treatment option for patients in a disease with high unmet need,” she said.

The FDA’s review of Orencia was part of Project Orbis, which “enabled concurrent and/or shared FDA review with the health authorities in Canada, Switzerland and as a pilot in Israel”, said BMS, although it stressed that Orencia was not yet approved for this indication in those countries.

Article by
Hugh Gosling

16th December 2021

From: Regulatory



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