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news.Alexion Pharmaceuticals has announced that the European Commission has granted an orphan drug designation to Soliris (eculizumab), a first-in-class terminal complement inhibitor, for the prevention of delayed graft function (DGF) after solid organ transplantation.
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DGF is an early and serious complication of organ transplantation that is characterized by the failure of a transplanted organ to function normally immediately following transplantation. In patients undergoing kidney transplantation, patients who develop DGF require dialysis in order to survive.
Soliris is currently approved in the US, EU, Japan and other countries for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), two debilitating, ultra-rare and life-threatening disorders caused by chronic uncontrolled complement activation.
Soliris is not approved in any country to prevent or treat DGF following kidney or other solid organ transplantation. Last month the US Food and Drug Administration (FDA) also granted an orphan drug designation to Soliris for the prevention of DGF in renal transplant patients.
Alexion executive vice president and R&D global head Dr Martin Mackay noted delayed graft function after transplantation is a debilitating and life-threatening condition because of the risk of losing the transplanted organ.
"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 improved clinical outcomes for transplant patients," Dr Mackay added.
The European Commission grants orphan medicinal product status to provide incentives to develop medicinal products to treat, prevent or diagnose diseases or conditions that affect no more than five in 10,000 persons in the EU.
The orphan medicinal product status designation would provide Alexion with certain benefits and incentives, including a period of market exclusivity if Soliris is approved in the EU to prevent or treat DGF.
Alexion plans to initiate a single multinational DGF registration study in kidney transplant patients with Soliris in 2014 to gather the clinical evidence needed to support approval for this indication.