Alexion Pharma's (ALXN) ALXN1007 Granted EC Orphan Drug Designation as GVHD Treatment

August 29, 2016 4:34 PM EDT

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Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) announced that the European Commission has granted orphan drug designation (ODD) to ALXN1007, a novel anti-inflammatory monoclonal antibody targeting complement protein C5a, for the treatment of 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

“Acute GI-GVHD is a devastating disease in which about one-third of severely affected patients die within the first six months following transplant despite best available care,” said Martin Mackay, Ph.D., Executive Vice President and Global Head of R&D at Alexion. “We are pleased that ALXN1007 has received orphan drug designation in the EU, recognizing the urgent need for an effective treatment for GVHD and the potential of ALXN1007 to improve response rates in patients facing this severe and life-threatening disorder.”

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. ALXN1007 is not approved in any country.

The European Commission grants orphan medicinal product status to provide incentives to develop medicinal products to treat, prevent or diagnose diseases or conditions that affect no more than five in 10,000 persons in the EU. The orphan medicinal product status designation provides Alexion with certain benefits and incentives in the EU, including a period of market exclusivity if ALXN1007 is approved to treat patients with acute GI-GVHD.



Jagasia M, Arora M, Flowers MED, et al. Risk factors for acute GVHD and survival after hematopoietic cell transplantation. Blood. 2012; 119(1):296-307.


MacMillan ML, DeFor TE, Weisdorf DJ. What predicts high risk acute graft-versus-host disease (GVHD) at onset?: identification of those at highest risk by a novel acute GVHD risk score. Br J Haematol. 2012;157:732-741.


Iqbal N, Salzman D, Lazenby AJ, Wilcox CM. Diagnosis of gastrointestinal graft-versus-host disease. Am J Gastroenterol. 2000 Nov;95(11):3034-8.

4. Weisdorf DJ, Snover DC, Haake R, et al. Acute upper gastrointestinal graft-versus-host disease: clinical significance and response to immunosuppressive therapy. Blood. 1990;76(3):624-629.
5. Bolaños-Meade J, Logan BR, Alousi AM, et al. Phase 3 clinical trial of steroids/mycophenolate mofetil vs steroids/placebo as therapy for acute GVHD: BMT CTN 0802. Blood. 2014;124(22):3221–3227.

Alexion Pharmaceuticals, Inc.


Stephanie Fagan, 475-230-3777

Senior Vice President, Corporate Communications


Kim Diamond, 475-230-3775

Executive Director, Corporate Communications



Elena Ridloff, CFA, 475-230-3601

Vice President, Investor Relations

Source: Alexion Pharmaceuticals, Inc.

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