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