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# FDA Orphan Designation for Alexion Drug Candidate
- URL: https://www.fdaweb.com/fda-orphan-designation-for-alexion-drug-candidate/
- Published: 2016-10-20T12:00:00.000Z
- Updated: 2026-09-14T21:41:22.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5137128

FDA has granted Alexion Pharmaceuticals an orphan drug designation for ALXN1007, a novel anti-inflammatory monoclonal antibody targeting complement protein C5a for treating acute graft-versus-host disease (GVHD). Alexion says it is currently investigating ALXN1007 in patients with acute GVHD in 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. Patients with severe, acute GI-GVHD have a 30% to 40% mortality rate within the first six months post-transplant. A Phase 2 trial is underway in patients with newly diagnosed acute GI-GVHD. More information on this trial is available at [www.clinicaltrials.gov](http://cts.businesswire.com/ct/CT?id=smartlink&url=http%3A%2F%2Fwww.clinicaltrials.gov&esheet=51442770&newsitemid=20161019006469&lan=en-US&anchor=www.clinicaltrials.gov&index=1&md5=d3929d741486968182c685d8666e8f6d&ref=fdaweb.com) under the identifier NCT02245412.