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Alexion Pharmaceuticals Inc announced that the EC as granted orphan drug designation (ODD) to ALXN1210, a highly innovative, longer-acting C5 antibody being evaluated in patients with paroxysmal nocturnal hemoglobinuria (PNH).
PNH is a debihlitating, 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).
“Patients with PNH are at continuous risk for severe and life-threatening consequences resulting from the ongoing complement-mediated destruction of red blood cells,” said Martin Mackay, Ph.D., Executive Vice President and Global Head of R&D at Alexion. “Soliris has been approved for the treatment of patients with PNH since 2007 and has dramatically changed the outlook for patients with this disease. Preliminary results from our ongoing clinical studies of ALXN1210, which has a half-life nearly three times that of Soliris, have shown rapid, complete, and sustained complement inhibition in treated patients with PNH. We are pleased that the Committee for Orphan Medicinal Products recognized that ALXN1210 has the potential to offer a meaningful clinical advantage for patients living with this devastating ultra-rare disorder.”
Alexion is evaluating ALXN1210 in a Phase 1/2 study and a Phase 2 study in patients with PNH. Both studies have exceeded target enrollment. More information on these clinical trials is available at www.clinicaltrials.gov under the identifiers NCT02598583 and NCT02605993. Alexion also plans to initiate a clinical program with ALXN1210 in patients with atypical hemolytic uremic syndrome (aHUS), another ultra-rare and life-threatening disease caused by chronic uncontrolled complement activation, in 2016. ALXN1210 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 ALXN1210 is approved to treat patients with PNH.
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 Soliris 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.1 PNH has been identified more commonly among patients with disorders of the bone marrow, including aplastic anemia (AA) and myelodysplastic syndromes (MDS).4,5,6 In patients with thrombosis of unknown origin, PNH may be an underlying cause.1
About ALXN1210
ALXN1210 is a highly innovative, longer-acting C5 antibody being evaluated by Alexion for the treatment of patients with PNH. In early studies, ALXN1210 has demonstrated rapid, complete, and sustained reduction of free C5 activity and a terminal half-life of more than 30 days, which may facilitate a monthly or longer dosing interval. Alexion is conducting two clinical studies of ALXN1210 in patients with PNH—a Phase 1/2 dose-escalating study and an open-label, multi-dose Phase 2 study.
About Soliris® (eculizumab)
Soliris is a first-in-class terminal complement inhibitor developed from the laboratory through regulatory approval and commercialization by Alexion. Soliris is approved in the U.S. (2007), European Union (2007), Japan (2010) and other countries as the first and only treatment for patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis. PNH is a debilitating, ultra-rare and life-threatening blood disorder, characterized by complement-mediated hemolysis (destruction of red blood cells). Soliris is also approved in the U.S. (2011), European Union (2011), Japan (2013) and other countries as the first and only treatment for patients with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy, or TMA (blood clots in small vessels). aHUS is a debilitating, ultra-rare and life-threatening genetic disorder characterized by complement-mediated TMA. Soliris is not indicated for the treatment of patients with Shiga-toxin E. coli-related hemolytic uremic syndrome (STEC-HUS). For the breakthrough medical innovation in complement inhibition, Alexion and Soliris have received some of the pharmaceutical industry’s highest honors: the Prix Galien USA (2008, Best Biotechnology Product) and France (2009, Rare Disease Treatment).
More information on Soliris, including the full U.S. prescribing information, is available at www.soliris.net.
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: www.alexion.com.
References
1. Socié G, Mary JY, de Gramont A, et al. Paroxysmal nocturnal haemoglobinuria: long-term follow-up and prognostic factors. Lancet. 1996: 348:573-577.2. Parker C, Omine M, Richards S, et al. Diagnosis and management of paroxysmal nocturnal hemoglobinuria. Blood. 2005;106(12):3699-3709.
3. Hillmen P, Lewis SM, Bessler M, Luzzatto L, Dacie JV. Natural history of paroxysmal nocturnal hemoglobinuria. N Engl J Med. 1995;333:1253-1258.
4. Wang H, Chuhjo T, Yasue S, Omine M, Naka S. Clinical significance of a minor population of paroxysmal nocturnal hemoglobinuria-type cells in bone marrow failure syndrome. Blood. 2002;100 (12):3897-3902.
5. Iwanga M, Furukawa K, Amenomori T, et al. Paroxysmal nocturnal haemoglobinuria clones in patients with myelodysplastic syndromes. Br J Haematol. 1998;102(2):465-474.
6. Maciejewski JP, Rivera C, Kook H, Dunn D, Young NS. Relationship between bone marrow failure syndromes and the presence of glycophosphatidyl inositol-anchored protein-deficient clones. Br J Haematol. 2001;115:1015-1022.
Contact:
Alexion Pharmaceuticals, Inc.
MediaStephanie Fagan, 203-271-8223
Senior Vice President, Corporate Communications
OR
Amanda Fahey, 203-699-7240Associate Director, Corporate Communications
OR
InvestorsElena Ridloff, CFA, 203-699-7722
Vice President, Investor Relations
