Biotech Drug Said to Be Useful Against a Rare Blood Ailment

A drug developed by a small biotechnology company has been found to be highly effective in treating a rare, often debilitating blood disorder that can cause life-threatening clots, the company is expected to announce today. The maker, Alexion Pharmaceuticals, said that its drug, eculizumab, sharply reduced the need for blood transfusions in a late-stage clinical trial involving patients with the disorder, called paroxysmal nocturnal hemoglobinuria or P.N.H. Alexion, based in Cheshire, Conn., said it planned to file for regulatory approval by the end of the year, after completing another trial focused on the drug's safety. Eculizumab would be the first product for Alexion, which was founded in 1992, and the first drug specifically to treat P.N.H. In that disease, red blood cells are destroyed by a person's own immune system, causing hemoglobin to spill into the bloodstream and be excreted in urine. The urine tends to turn dark overnight but clears up during the day. People with P.N.H. can have life-threatening blood clots, anemia, severe abdominal pain, difficulty swallowing and erectile dysfunction. In the clinical trial, about half the patients given the drug had stable hemoglobin levels over six months without needing a transfusion, researchers say. None of those getting a placebo had such results. But the trial, involving 87 patients, was too small and short to determine if the drug prevented blood clots, the leading cause of death from the disease. The lead investigator in the trial, Dr. Peter Hillmen, said eculizumab had allowed some patients to return to normal activities. "We've got patients who had been on morphine continuously beforehand," said Dr. Hillmen, a hematologist in Leeds, England, "and have stopped the morphine and have started businesses." Alexion will probably charge $100,000 to $200,000 a year for the drug, Leonard Bell, the chief executive, said. The results could help Alexion recover from a setback in November, when its similar drug failed in a clinical trial to significantly reduce the rate of heart attacks after coronary-bypass surgery. The stock, which traded as high as $30 in September, plunged below $19. Yesterday, it closed at $21.95, down 6 cents. Alexion estimates that 8,000 to 10,000 people in North America and Western Europe have P.N.H. The company for years resisted entreaties from Dr. Hillmen, an expert on P.N.H., to test its drug as a treatment for that disease, preferring to focus on disorders like rheumatoid arthritis. But eculizumab did not work well enough against arthritis to compete in a crowded field of products. And Alexion came to realize that drugs for rare diseases can be very lucrative if their price is high enough. P.N.H. is caused by a genetic mutation that arises during a person's lifetime and leaves blood cells vulnerable to attack by part of the immune system known as the complement system. Eculizumab, which is given every two weeks by infusion, is a monoclonal antibody that blocks part of the complement system. Inhibition of the complement system could leave people vulnerable to infections, particularly meningitis. In the trial, patients given the drug did not have a higher infection rate than those getting the placebo, Dr. Bell said, though all the patients had received meningitis vaccinations. "Source":[ http://www.nytimes.com/2006/01/26/business/26drug.html?_r=1&oref=slogin]

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