Pharmaceutical Business review

Alexion’s Soliris gets orphan drug status in Europe for organ transplantation

The company said that graft rejection can cause severe injury to the transplanted organ and is a significant barrier to successful transplantation.

Currently, the drug is approved in the US, EU, Japan and other countries to treat 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.

The company 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.

The drug is not approved in any country to prevent or treat rejection following kidney or other solid organ transplantation.

Alexion executive vice president, global head of R&D Martin Mackay said 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.

"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," Mackay said.