Alexion's ALXN1007 granted orphan drug designation from FDA
Alexion Pharmaceuticals announced that the U.S. Food and Drug Administration has granted orphan drug designation to ALXN1007, a novel anti-inflammatory monoclonal antibody targeting complement protein C5a, for the treatment of acute graft-versus-host disease. Alexion is currently investigating ALXN1007 in patients with acute GVHD of the lower gastrointestinal tract, a severe and life-threatening rare autoimmune disease that can occur as a complication of stem cell or bone marrow transplantation. "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. "Alexion is pleased that the FDA has granted orphan drug designation to ALXN1007, recognizing its potential to improve response rates in patients facing acute GI-GVHD." ALXN1007 is currently being evaluated in a Phase 2 study in patients with newly diagnosed acute GI-GVHD.