Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) today announced the presentation of new data demonstrating the long-term benefits of Soliris ® (eculizumab) in patients with paroxysmal nocturnal hemoglobinuria (PNH), a debilitating, ultra-rare and life-threatening blood disorder characterized by complement-mediated hemolysis (destruction of red blood cells). Data including the clinical benefits of Soliris in the treatment of patients with PNH and atypical hemolytic uremic syndrome (aHUS) were presented at the 54th Annual Meeting of the American Society of Hematology (ASH) in Atlanta:
- Ten-year data from a large cohort of patients with PNH from the United Kingdom (UK) confirm the long-term safety and efficacy of chronic Soliris therapy and demonstrate the impact of Soliris on quality of life. 1
- The long-term safety of Soliris was also demonstrated in PNH clinical development trials in which patients were treated continuously with Soliris and followed for up to 5.5 years. 2
- Data from an international PNH registry showed that Soliris significantly reduced the risk of thromboembolism (TE) in patients with PNH. 3
- Researchers from South Korea presented data confirming that hemolysis is an independent risk factor for TE in patients with PNH. 4
- Researchers in Japan observed a rare genetic polymorphism in the terminal complement protein C5 in Japanese patients with PNH who had no or minimal reduction in lactate dehydrogenase (LDH) while on Soliris therapy (9 out of 250 treated, or 3.6%). 5
- The ASH meeting also featured presentations of two-year data highlighting the long-term benefits of chronic Soliris therapy in patients with aHUS, 6,7 an ultra-rare, genetic disease characterized by complement-mediated thrombotic microangiopathy (TMA), the formation of blood clots in small vessels throughout the body.
Soliris, a first-in-class terminal complement inhibitor, specifically targets uncontrolled complement activation, and is approved in the United States, European Union, Japan and other countries as the first and only treatment for patients with PNH. Soliris is also approved in the US and EU as the first and only treatment for patients with aHUS.