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news.Alexion Pharmaceuticals has completed enrollment in a phase III trial of its drug eculizumab in patients with a rare blood disorder known as paroxysmal nocturnal hemoglobinuria, or PNH.
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PNH is characterized by the onset of severe anemia, chronic fatigue and intermittent episodes of dark colored urine. Estimates suggest that 2,000 – 10,000 people in the US, and a similar number in Europe, suffer from this disease.
Eculizumab has been granted orphan drug status from both the US and European regulatory agencies to treat PNH. If approved, eculizumab would represent the first drug from a new class of anti-inflammatory therapeutics, known as terminal complement inhibitors, as well as the first drug available specifically for patients suffering from PNH.
Approximately 95 PNH patients have been enrolled into the trial, which will include 12 months of treatment with a six month interim analysis.
“This is another milestone in the program and with the two phase III trials having completed enrollment, we remain focused on driving the PNH program forward to meet the needs of the underserved PNH patient population,” said Dr Leonard Bell, chief executive officer of Alexion.