Safe Effective Therapy Treats Paroxysmal Nocturnal Hemoglobinuria

A therapy has been developed for paroxysmal nocturnal hemoglobinuria (PNH), a rare, disabling and life-threatening blood disorder defined by chronic red blood cell destruction, or hemolysis.

Hemolysis can cause one or more of the following symptoms in patients with PNH: severe anemia, disabling fatigue, recurrent pain, shortness of breath, pulmonary hypertension, intermittent episodes of dark colored urine (hemoglobinuria), kidney disease, impaired quality of life, and blood clots (thromboses). PNH often strikes people in the prime of their lives, with an average age of onset in the early1930's. The estimated median survival for PNH patients is between 10 and 15 years from the time of diagnosis.

PNH is an acquired genetic blood disorder defined by hemolysis, in which patients' red blood cells are destroyed by complement, a component of the body's immune system. PNH is a rare disease that affects an estimated 8,000 to 10,000 people in North America and Europe. Ten percent of all patients first develop symptoms at 21 years of age or younger. PNH develops without warning and can occur in men and women of all races, backgrounds, and ages.

Alexion Pharmaceuticals, Inc., (Cheshire, CN, USA) has received marketing approval from the U.S. Food and Drug Administration (FDA) for Soliris (eculizumab) for treatment of patients with PNH to reduce hemolysis. Patients with PNH are missing a specific protein that normally protects red blood cells from destruction by a component of the immune system called terminal complement. Soliris, a complement inhibitor prevents hemolysis by selectively blocking terminal complement.

PNH often strikes people in the prime of their lives, with an average age of onset in the early 30's. The estimated median survival for PNH patients is between 10 and 15 years from the time of diagnosis.

Studies showed that Soliris reduced hemolysis in every treated patient. The reductions in hemolysis occurred within one week of initiating treatment and were sustained for periods of up to 54 months with continued dosing of Soliris. The reduction in hemolysis expanded the number of circulating PNH cells and, thereby, increases the hemoglobin level. Soliris patients reported less fatigue and improved health-related quality of life. There were fewer thrombotic events with Soliris treatment than during the same period before treatment.

"Soliris brings real hope to people who live daily with the devastating effects of PNH. With the approval of Soliris, we now have a therapy that dramatically improves the lives of patients suffering from this disease. Importantly, all patients with this life-threatening disease will be eligible for treatment,” said Leonard Bell, M.D., CEO of Alexion Pharmaceuticals.

"Soliris directly targets the underlying disease process responsible for debilitating symptoms that may contribute to shortened life spans of PNH patients,” said Wendell F. Rosse, M.D., emeritus professor of medicine at Duke University. "Having cared for more than 300 patients with PNH over my career, I believe this is the most important advance that has been made in the treatment of this disease. Treatment with Soliris markedly decreases the hemolysis responsible for anemia, fatigue, poor patient functioning, and blood clots in PNH patients.”


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