Alexion Pharma (ALXN) Reports FDA Orphan Drug Designation for ALXN1007 to Treat GVHD

October 19, 2016 4:34 PM EDT

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Alexion Pharmaceuticals, Inc. (NASDAQ: ALXN) today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to ALXN1007, a 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 that can occur as a complication of stem cell or bone marrow transplantation.1,2,3

“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. More information on this trial is available at under the identifier NCT02245412. In August of 2016, ALXN1007 was granted ODD by the European Commission. ALXN1007 is not approved in any country.

The FDA, through its Office of Orphan Products Development (OOPD), grants orphan status to drugs and biologic products that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. ODD provides a drug developer with certain benefits and incentives, including a period of marketing exclusivity if regulatory approval is ultimately received for the designated indication.

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