New Treatment For Life-Threatening Disorder (NAPSA)—Scientists have succeeded in finding a “needle in the haystack” of medical treatment that brings hope to patients desperately awaiting solutions to a life- threatening disease. The Food and Drug Administration has now approved thefirst treatment for a rare blood disorder: paroxysmal nocturnal hemoglobinuria (PNH). PNH results from chronic red blood cell destruction that leads to severe anemia, disabling fatigue and recurrent pain. It develops without warning in men and womenof all races, backgrounds and ages. The average length of survival is between 10 and 15 years from the timeof diagnosis. The new treatment works by selectively blocking a component of the body’s immune system from attacking red blood cells, thereby reducing symptoms. Until now, patients could only managetheir symptoms through blood transfusions, immunosuppressive ther- apy and, infrequently, bone marrow transplantations—a high-risk and painful last-resort procedure. “The Aplastic Anemia & MDS International Foundation [AA& MDSIF' is extremely pleased that PNH patients now havea treatment specifically for their disease. Doctors now have a wayto help people with a rare but life-threat- ening disease. chairperson and the motherof a child with aplastic anemia. Alexion, the company that cre- ated the new drug, Soliris (eculi- zumab), has also introduced Soliris OneSource, a treatment support service for PNH patients and their health care providers. Each patient in the program gets support from a registered nurse case manager who provides information and facilitates solutions to help patients. The company’s goal is that all PNH patients who can benefit from Soliris will have accesstoit. You can learn more about OneSource by calling (888) SOLIRIS This is a tremendousstep forward (888-765-4747) or visiting www. soliris.net. More information for anyone with bone marrowfailure,” said Sherrie Van Vliet, vice- eases is at www.rarediseases.org and www.aamds.org. for all who suffer from PNH—and about PNH and other rare dis- none eee n eee n nen nee n een eee DL eee eee eee eee eee Note to Editors: Important Safety Information: Soliris is generally well tolerated. The most frequent adverse events observed in clinical studies were headache, nasopharyngitis (a runny nose), back pain and nausea. Treatment with Soliris should not alter anticoagulant management becausethe effect of withdrawal of anticoagulant therapy during Soliris treatment has not been established. The product label for Soliris also includes a boxed warning: “Soliris increases the risk of meningococcal infections. Vaccinate patients with a meningococcal vaccine at least 2 weeks priorto receiving the first dose of Soliris; revaccinate according to current medical guidelines for vaccine use. Monitor patients for early signs of meningococcal infections, evaluate immediately if infection is suspected, and treat with antibiotics if necessary.” Two out of 196 vaccinated PNH patients treated with Soliris experienced a serious meningococcal infection. Prior to beginning Soliris therapy, all patients and their prescribing physicians will be enrolled in the Soliris Safety Registry, Which is part of a special risk management program that involves initial and continuing education and long-term monitoring for detection of new safety findings. See full prescribing information at www.soliris.net.
