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Hemoglobinuria, Paroxysmal

Patient Medical Areas

June 19, 2006

Alexion Pharmaceuticals has announced positive interim results of a phase III trial, dubbed SHEPHERD of Soliris (eculizumab) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). 6-month data met their primary surrogate efficacy endpoint, significantly reducing median lactate dehydrogenase levels (a biomarker for intravascular hemolysis) by 87%, from 2051 U/l at baseline to 270 U/l at 26 weeks (p<<0.00000000001). Significance was also achieved in all pre-specified secondary endpoints, including reduction in mean LDH change from baseline and quality of life as measured by the Functional Assessment of Chronic Illness Therapy Fatigue (FACIT-Fatigue) diagnostic measure. The most frequent adverse events associated with Soliris treatment were headache, nasopharyngitis, and nausea. This open-label study enrolled 97 PNH patients across 33 sites in the US, Canada, Europe and Australia, who were to receive treatment with the drug for 12 months.