Alexion Pharmaceuticals Inc. announced that the European Commission has granted an orphan drug designation (ODD) to Soliris (eculizumab), a first-in-class terminal complement inhibitor, for the prevention of graft rejection following solid organ transplantation. Graft rejection can cause severe injury to the transplanted organ and is a significant barrier to successful transplantation.

Soliris is currently approved in the United States, European Union, 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. Alexion is investigating Soliris for the prevention of acute antibody-mediated rejection (AMR) in kidney transplant recipients, and for the prevention of delayed graft function (DGF) in patients receiving deceased donor kidney transplants. Soliris is not approved in any country to prevent or treat rejection following kidney or other solid organ transplantation.

“Rejection after transplantation is a severe and potentially devastating occurrence for patients undergoing organ transplantation due to the very real risk of losing the transplanted organ,” said Martin Mackay, executive vice president, global head of R&D at Alexion. “By specifically inhibiting the terminal complement pathway, Soliris has the potential to lower the risk of rejection, a benefit that could lead to improved clinical outcomes for these patients.”

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 marketing exclusivity if Soliris is approved in the EU for the orphan therapeutic indication of acute antibody-mediated rejection (AMR).