Alexion Pharmaceuticals, Inc. (Nasdaq:ALXN) today announced that researchers will present data from clinical studies of Soliris ® (eculizumab) as a treatment for patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), two life-threatening and ultra-rare diseases caused by chronic uncontrolled complement activation, at the 55th Annual Meeting of the American Society of Hematology (ASH). Soliris is the first and only approved treatment for PNH and aHUS. Abstracts summarizing these data are published on the ASH website and can be accessed using the links below. The ASH annual meeting will be held December 7-10, 2013, at the Ernest N. Morial Convention Center in New Orleans.
Soliris was first approved in 2007 and is now approved in nearly 50 countries as a treatment for patients with PNH, 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 United States (2011), European Union (2011), Japan (2013) and other countries as a treatment for patients with aHUS, a genetic, chronic and ultra-rare disease associated with vital organ failure and premature death.
Soliris and PNH
The following abstract will be presented in a poster session on Saturday, December 7, 2013 from 5:30 – 7:30 p.m., Central Standard Time (CST):Abstract 1241: “Baseline Assessment of GPI-anchored Protein Deficient Blood Cells in Patients with Bone Marrow Failure (The OPTIMA study),” Obara, et al. Accessible at: https://ash.confex.com/ash/2013/webprogram/Paper63472.html The following abstracts will be presented in a poster session on Monday, December 9, 2013 from 6:00 – 8:00 p.m., Central Standard Time (CST): Abstract 3715: “Periodic Evaluation of the Clone Size is Mandatory in PNH: A Study of the Spanish Cohort of the International PNH Registry,” Villegas, et al. Accessible at: https://ash.confex.com/ash/2013/webprogram/Paper60116.html Abstract 3720: “Clinical Signs and Symptoms in Non-transfused Patients with Paroxysmal Nocturnal Hemoglobinuria from a Korean Prospective PNH Registry,” Lee, et al.