Bristol-Myers Squibb (BMS) has secured breakthrough therapy status from the US Food and Drug Administration (FDA) for its Orencia (abatacept) to prevent moderate to severe acute graft-versus-host disease (GvHD) in hematopoietic stem cell transplants from unrelated donors.
GvHD is a potentially life-threatening medical complication, which can affect patients receiving such transplants to treat certain genetic diseases and haematologic cancers.
Orencia is an immunomodulator that disturbs the continuous cycle of T–cell activation, which indicates rheumatoid arthritis (RA).
The designation was provided based on the results from an investigator-initiated trial supported by the company.
The phase 2 study evaluated the impact of Orencia on the prevention of severe acute GvHD, when combined with a standard GvHD prophylactic regimen administered to patients with hematologic malignancies receiving a stem cell transplant from an unrelated, HLA-matched or mismatched donor.
Orencia was approved in the US to reduce 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.
It was also approved to reduce signs and symptoms in patients two years of age and older with moderately to severely active polyarticular juvenile idiopathic arthritis. In addition, Orencia was approved to treat adult patients with active psoriatic arthritis.
Bristol-Myers Squibb Orencia development lead Dr Brian Gavin said: “We are excited about the potential of ORENCIA to improve outcomes for patients receiving unrelated stem cell transplants.