Alexion Pharmaceuticals has received orphan drug designation from the US Food and Drug Administration (FDA) for its terminal complement inhibitor Soliris (eculizumab), intended for the prevention of delayed graft function (DGF) in renal transplant patients.
DGF is an early and serious problem of organ transplantation that is characterised by the failure of a transplanted organ to function normally immediately after transplantation.
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The company said that in patients undergoing a kidney transplant, DGF leads to the patient requiring dialysis in order to survive.
Currently, Soliris is approved to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS).
PNH and aHUS are two debilitating, ultra-rare and life-threatening disorders caused by chronic uncontrolled complement activation.
The drug is not approved in any country to prevent or treat DGF following kidney or other solid organ transplantation.
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By GlobalDataAlexion executive vice-president and global head of R&D Martin Mackay said for kidney transplant patients with increased risk, there is a serious unmet medical need for a treatment to prevent delayed graft function and its harmful consequences.
“By specifically inhibiting the terminal complement pathway, which is believed to play a critical role in the development of DGF, Soliris has the potential to lower the risk of DGF, a benefit that may have positive implications for longer-term kidney function and clinical outcomes for patients,” Mackay said.
“In addition, a significant number of donor kidneys are reportedly never used and thus discarded each year due to the risk of poor outcomes associated with DGF, therefore reducing the risk of DGF may enable more patients to receive a kidney transplant.”
The company intends to start a single multinational DGF registration trial with Soliris later in 2014 and also seeks to work closely with the FDA to gather the clinical evidence needed to support approval for this indication.
Image: Kidney location after transplantation. Photo: courtesy of Adrian J Hunter.
