Alexion's eculizumab shows efficacy in treating paroxysmal nocturnal hemoglobinuria, study says

By Angela McDaniels

Seattle, Dec 12 - Alexion Pharmaceuticals Inc. said preliminary trial results show that paroxysmal nocturnal hemoglobinuria patients given eculizumab have shown sustained and significant reductions in red blood cell destruction and in the need for blood transfusions.

Seven of the 10 patients treated with eculizumab alone, or in combination with erythropoeitin in aplastic patients became transfusion independent for at least the last 17 months of the study, the company said.

The results also showed a sustained and stable increase in paroxysmal nocturnal hemoglobinuria red blood cell counts and that the drug is well tolerated.

The 10-patient open-label extension program of an initial pilot trial is being conducted in the United Kingdom at two sites. All patients have now completed at least three years of chronic eculizumab therapy.

Following the initial 12-week pilot trial, all 11 patients chose to participate in two consecutive one-year extension studies, the first of which was published in the Oct. 1 issue of the journal Blood. Ten of the original 11 patients enrolled into a third one-year extension study and continue on eculizumab.

The results were presented by investigators from Leeds Teaching Hospital, St. George's Hospital and Alexion at the 47th Annual American Society of Hematology Meeting and Exposition in Atlanta on Saturday.

"In anticipation of pivotal paroxysmal nocturnal hemoglobinuria trial data early next year, we are keenly focused on progressing our internal efforts to compile our regulatory applications for eculizumab in paroxysmal nocturnal hemoglobinuria and to advance our commercialization preparations both in the United States and Europe," chief executive officer Leonard Bell said in a company news release.

Eculizumab is a monoclonal antibody drug that blocks the terminal complement pathway of the immune system. Terminal complement has been linked to the destruction of red blood cells, or hemolysis, characteristic of paroxysmal nocturnal hemoglobinuria , the company said.

Eculizumab has been granted orphan drug status for treating paroxysmal nocturnal hemoglobinuria from both U.S. and European regulatory agencies. If approved, eculizumab would represent the first drug from a new class of anti-inflammatory therapeutics - terminal complement inhibitors - and the first drug available specifically for patients suffering from paroxysmal nocturnal hemoglobinuria.

Paroxysmal nocturnal hemoglobinuria is a blood disorder characterized by the onset of severe anemia, chronic fatigue and intermittent episodes of dark-colored urine. Paroxysmal nocturnal hemoglobinuria patients are also at increased risk of forming life-threatening blood clots, or thromboses, which are a leading cause of death in this disease.

Alexion is based in Cheshire, Conn., and develops therapeutic products aimed at treating patients with a wide array of severe disease states, including hematologic and cardiovascular disorders, autoimmune diseases and cancer.

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