Eculizumab demonstrates its efficacy and safety in different populations of patients with paroxysmal nocturnal Paroxistica

in the seventeenth European Hematology Congress (EHA)

– according to the results of different studies in different populations of patients with HPN, eculizumab inhibits the uncontrolled complement activation and intravascular hemolysis.

Barcelona, July of 2012. Treatment with eculizumab demonstrates its efficacy and safety in different populations of patients who suffer from Hemoglobinuria Paroxistica night (HPN), a disease ultra-rara, progressive, which consists of uncontrolled complement system activation and that is life-threatening, as shown in studies presented at the seventeenth European Hematology Congress (EHA)held in Amsterdam.

In this context, it should be noted between the communications presented at the Congress, the effectiveness of eculizumab in patients who, despite having a history of thrombosis, hemolysis was not severe enough to require blood transfusions. In fact, in a particular case that had suffered three major thrombotic crises for a period of five years, after the administration of eculizumab patient has not experienced relapse.

Also, found a reduction of 86% of hemolysis with eculizumab in patients with HPN, valued by the LDH to 4.5 years. These data are very important since chronic complement-mediated hemolysis is the underlying cause of diseases associated with the HPN and its subsequent mortality.

Patients with hemolysis 1.5 above the limit of normal (as measured by the LDH) at the time of the diagnosis had a mortality rate 5 times greater than the general population of similar age and sex (P < 0.001). That is why early diagnosis is essential to improving treatment and the prognosis of these patients.

Eculizumab is the only drug approved by the FDA (Food and Drug Administration) and the EMEA (European Medicines Agency) in 2007, has shown to dramatically reduce the risk of thrombosis in the HPN patients, improving their quality of life and comparing their survival to that of the general population.

Paroxysmal nocturnal

Paroxysmal nocturnal hemoglobinuria (PNH) is a ultra-rara disease that affects about 250 patients in Spain. It is a genetic disorder in which certain complement inhibitory proteins do not adhere properly to the red blood cells and, consequently, these remain unprotected action lithic complement (primary immune) system and are destroyed prematurely and Chronicle (a process called hemolysis).2 this systemic disease deteriorate progressively and severe patientsdramatically increasing the risk of thrombosis, this being the main cause of death along with inadequate renal.3-4

La HPN is a disease of difficult diagnosis, which normally Debuts around 30 years. 4 the HPN patients median survival ranges between 10 and 15 years since his diagnosis, an early diagnosis and appropriate treatment is essential to improve the prognosis of these patients, especially when thrombosis, abdominal pain, chest pain or disnea2, 5 6

before the advent of eculizumab, there was no specific treatment of the HPN, having only symptomatic such as blood transfusions and immunosuppressive therapy remedial measures unspecific. 1

informationAlexion on

Alexion is a biotechnology company with a global presence that researches and develops innovative therapies for patients with ultra-raras diseases, which are very rare, debilitating and life-threatening. Alexion was founded in 1992 and currently employs more than 800 people throughout the world committed to the discovery and access to new drugs for hematologic, kidney and neurological diseases, as well as for cancer, autoimmune diseases and rejection in transplantation.

Alexion was established in Europe in 2005 and has expanded its presence across the continent. Alexion won the Manuel United States Award for best biotechnology product in 2008. Later, he obtained the Manuel France Award in 2009 for having the most innovative drug in the category of rare diseases.

5 Lee JW, Jang JH, Lee JH, et to the. High prevalence and mortality associated with thromboembolism in Asian patients with PNH. Haematologica. 2010; 95 (s2): Abstract 0505.