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news.Alexion Pharmaceuticals' drug Soliris was shown to be effective and safe after six months of a phase III trail evaluating the drug in a rare form of anemia according to preliminary data reported by the company.
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The 12 month study is focused on examining safety as well as efficacy of the drug in patients with paroxysmal nocturnal hemoglobinuria (PNH), an acquired genetic blood disorder characterized by destruction of red blood cells by the body’s complement system (a component of the immune system).
Patients with PNH lack naturally-occurring complement inhibitors which normally prevent red blood cell destruction. Soliris, a long-acting C5 terminal complement inhibitor, is a monoclonal antibody drug that selectively blocks terminal complement activation. There currently is no approved therapy specifically available for treatment of PNH.
The interim results show that Soliris appeared to be safe and well tolerated. All pre-specified primary and secondary efficacy endpoints in were achieved with statistical significance.
The pre-specified primary surrogate of efficacy endpoint was intravascular hemolysis, the underlying disease process and primary clinical manifestation in PNH, as measured by lactate dehydrogenase area under the curve (LDH AUC). The LDH AUC was significantly decreased; LDH was reduced by 87% from a median of 2051 at baseline to 270 U/L after 26 weeks. The most frequent adverse events with Soliris were headache, nasopharyngitis, and nausea.