The FDA has granted orphan drug designation to ALXN1007, Alexion’s novel anti-inflammatory monoclonal antibody targeting complement protein C5a, for the treatment of acute graft-versus-host disease (GVHD).

Alexion is currently investigating ALXN1007 in patients with acute GVHD of the lower gastrointestinal tract (GI-GVHD), a severe and life-threatening rare autoimmune disease.

“There is an urgent need for an effective treatment for GI-GVHD, which causes severe symptoms and often leads to mortality following allogeneic hematopoietic stem cell transplantation. Currently, even with best available care, about one-third of severely affected patients die from this devastating complication within six months of onset,” said Martin Mackay, Ph.D., Executive Vice President and Global Head of R&D at Alexion.