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Alexion Joins Eurordis, NORD And Patient Organizations Worldwide In Celebrating Rare Disease Day 2013

Alexion developed Soliris ® (eculizumab), a first-in-class terminal complement inhibitor, from the laboratory through regulatory approval and commercialization. Soliris is approved in the US, European Union, Japan and other countries as the first and only treatment for patients with paroxysmal nocturnal hemoglobinuria (PNH), a debilitating, life-threatening and ultra-rare blood disorder. Soliris is also approved in the US and the European Union as the first and only treatment for patients with atypical hemolytic uremic syndrome (aHUS), a debilitating and life-threatening ultra-rare genetic disorder.

“We understand that every day is Rare Disease Day for patients and families who suffer from severe and life-threatening ultra-rare disorders and often live without hope because an effective treatment option is not available,” said Leonard Bell, M.D., Chief Executive Officer of Alexion. "The employees of Alexion are committed to developing and delivering therapies that can transform the lives of these patients. We now serve patients in 50 countries by focusing on disease education to help patients with PNH and aHUS receive an accurate diagnosis and appropriate treatment. At the same time, we continue to invest in research and development with the goal of providing highly innovative therapies to patients with additional severe and life-threatening disorders, which also happen to be extremely rare.”

Bringing Hope Across the Globe

Alexion is currently developing five highly innovative therapeutics, including eculizumab (Soliris ®), which are being investigated in nine severe and life-threatening ultra-rare disorders. The company’s development programs are solely focused on:

  • Severe disorders with devastating and life-threatening medical consequences
  • Disorders with ineffective, or no treatment options
  • Disorders that are ultra-rare and affect very small numbers of patients

To learn more about Rare Disease Day, visit  www.rarediseaseday.us for U.S. activities and www.rarediseaseday.org for global activities.

About Rare and Ultra-Rare Disorders

In the United States, a disease is defined as rare if it affects fewer than 650 patients per million of population. 2 The European Union definition of a rare disease is one that affects fewer than five patients per 10,000 of population. 3 In contrast, a disease is generally considered to be ultra-rare if it affects fewer than 20 patients per million of population 4 (one patient per 50,000) – and most ultra-rare diseases affect far fewer people than this.

Despite the very small numbers of patients they affect, the impact of these rare and ultra-rare diseases on patients, their families, and society is profound, as many are severe, chronic and progressive, with high mortality rates. Patients with severe and life-threatening ultra-rare diseases often live without hope, have no effective treatment options and may face premature death.

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