Alexion Says FDA Grants Orphan Drug Designation To ALXN1210


Alexion Pharmaceuticals Inc announced that the U.S. FDA has granted orphan drug designation (ODD) to ALXN1210, a highly innovative, longer-acting anti-C5 antibody that inhibits terminal complement, which is being evaluated for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH).

PNH is a debilitating, ultra-rare, life-threatening blood disorder in which uncontrolled activation of complement, a component of the immune system, results in hemolysis (destruction of a patient’s red blood cells).1

“Alexion is committed to achieving the highest levels of innovation to address the needs of patients suffering from PNH, a devastating ultra-rare disorder,” said Martin Mackay, Ph.D., Executive Vice President and Global Head of R&D at Alexion. “We are pleased that the FDA has recognized the potential for ALXN1210 to offer a significant therapeutic advantage for patients with PNH. Data from our ongoing clinical studies have shown rapid, complete, and sustained complement inhibition in treated patients, and we look forward to continuing to evaluate this highly innovative molecule in our Phase 3 trial of ALXN1210 administered every eight weeks.”

Alexion is currently enrolling patients in Phase 3 trials of ALXN1210 in patients with PNH as well as in patients with atypical hemolytic uremic syndrome (aHUS), another ultra-rare and life-threatening disease caused by chronic uncontrolled complement activation. More information on these clinical trials is available at under the identifiers NCT02946463 and NCT02949128. In June 2016, ALXN1210 was granted ODD by the European Commission for the treatment of patients with PNH. ALXN1210 is protected by a composition of matter patent in the U.S. and Europe through 2035. ALXN1210 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.

About Paroxysmal Nocturnal Hemoglobinuria (PNH)
PNH is an ultra-rare blood disorder in which chronic, uncontrolled activation of complement, a component of the normal immune system, results in hemolysis (destruction of the patient's red blood cells). PNH strikes people of all ages, with an average age of onset in the early 30s.1 Approximately 10 percent of all patients first develop symptoms at 21 years of age or younger.2 PNH develops without warning and can occur in men and women of all races, backgrounds and ages. PNH often goes unrecognized, with delays in diagnosis ranging from one to more than 10 years.3 In the period of time before treatment was available, it had been estimated that approximately one-third of patients with PNH did not survive more than five years from the time of diagnosis.4 PNH has been identified more commonly among patients with disorders of the bone marrow, including aplastic anemia (AA) and myelodysplastic syndromes (MDS).5,6,7 In patients with thrombosis of unknown origin, PNH may be an underlying cause.8

About ALXN1210
ALXN1210 is a highly innovative, longer-acting anti-C5 antibody discovered and developed by Alexion that inhibits terminal complement. In early studies, ALXN1210 demonstrated rapid, complete, and sustained reduction of free C5 levels.9 Alexion has completed enrollment in two ongoing clinical studies of ALXN1210 in patients with PNH—a Phase 1/2 dose-escalating study and an open-label, multi-dose Phase 2 study that is also evaluating longer dosing intervals beyond 8 weeks.

ALXN1210 is currently in Phase 3 trials in patients with PNH and aHUS. In addition, Alexion is conducting a Phase 1 study to evaluate a new formulation of ALXN1210 administered subcutaneously in healthy volunteers.In June 2016, the European Commission granted Orphan Drug Designation (ODD) to ALXN1210 for the treatment of patients with PNH.

About Alexion
Alexion is a global biopharmaceutical company focused on developing and delivering life-transforming therapies for patients with devastating and rare disorders. Alexion is the global leader in complement inhibition and has developed and commercializes the first and only approved complement inhibitor to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), two life-threatening ultra-rare disorders. In addition, Alexion’s metabolic franchise includes two highly innovative enzyme replacement therapies for patients with life-threatening and ultra-rare disorders, hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D). Alexion is advancing the most robust rare disease pipeline in the biotech industry with highly innovative product candidates in multiple therapeutic areas. This press release and further information about Alexion can be found at:


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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


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