The European Commission has granted an orphan drug designation to Alexion Pharmaceuticals’ Soliris (eculizumab) to prevent graft rejection in organ transplants.

The move will 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).

“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 biopharma company is also 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.

Considered to be possibly the most expensive drug available, the first-in-class terminal complement inhibitor has already been approved for ultra-orphan diseases in the USA, EU, Japan and other countries for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). In the UK, the drug as a treatment for aHUS commands a cost in the region of £340,200 per adult patient per year. As a result, it has struggled to be reimbursed.

Alexion's breakthrough approach in terminal complement inhibition has received the pharmaceutical industry's highest honors: the 2008 Prix Galien USA Award for Best Biotechnology Product with broad implications for future biomedical research and the 2009 Prix Galien France Award in the category of Drugs for Rare Diseases.