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news.Alexion Pharmaceuticals has completed the process of screening and randomization of patients in a phase III trial of its drug eculizumab, to be used in the treatment of patients with PNS, a rare blood disorder.
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The study will now enter a six-month treatment phase with 85 patients randomized to receive either eculizumab or placebo, with the final patient anticipated to complete the treatment phase near the end of this year.
Eculizumab is a monoclonal antibody drug that seeks to address the deficiency of natural inhibitors in PNS sufferer’s blood cells.
There currently is no drug specifically available for treatment of patients with PNH, whose blood cells are deficient in natural inhibitors of terminal complement. Estimates suggest that up to 2,000 – 10,000 people in the US, and a similar number in Europe, suffer from this disease.
“We remain focused on driving the PNH program forward to meet the needs of the severely underserved PNH patient population,” said Dr Leonard Bell, CEO of Alexion.