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| |  Eculizumab Approved in Canada for Paroxysmal Nocturnal Haemoglobinuria (PNH) NEW YORK -- January 29, 2009 -- Health Canada has approved eculizumab (Soliris) for the treatment of paroxysmal nocturnal haemoglobinuria (PNH). Eculizumab is the first therapy approved in Canada for this disease. Eculizumab "has had a life-changing impact on patients with PNH," says Loree M. Larratt, MD, Department of Medicine, Divisional Director Clinical Hematology, University of Alberta at Edmonton, Edmonton, Alberta, Canada, one of the researchers who participated in eculizmumab clinical trials. Dr. Larratt says that haemolysis underlies the significant morbidities and mortality of PNH and that eculizumab reduced haemolysis "in every patient treated in clinical studies." Eculizumab was approved under Priority Review by Health Canada's Biologics and Genetic Therapies Directorate (BGTD). The marketing application submitted to the BGTD included safety and efficacy data from 3 multinational clinical studies in patients with PNH: (1) TRIUMPH, a placebo-controlled, 26-week, phase 3 study (N = 87); (2) SHEPHERD, an open-label, 52-week, phase 3 trial (N = 97 patients); and (3) E05-001, a long-term extension study. Eculizumab was approved by the US Food and Drug Administration and the European Commission in 2007 using data from the same studies and is currently being used to treat patients with PNH in the United States and more than 15 additional countries. "We are very excited that Health Canada has been able to move quickly to approve eculizumab. We have been hearing from Canadian patients affected by PNH who have been waiting for access to this innovative drug," said Durhane Wong-Rieger, PhD, Canadian Organization for Rare Disorders. Prior to approval of eculizumab in the United States and European Union, there were no therapies specifically available for the treatment of PNH, which was limited to symptom management through periodic blood transfusions, nonspecific immunosuppressive therapy, and infrequently bone marrow transplantations. Eculizumab is generally well tolerated. The most frequent adverse events observed in clinical studies were headache, nasopharyngitis, back pain, and nausea. Treatment with eculizumab should not alter anticoagulant management, because the effect of withdrawal of anticoagulant therapy during eculizumab treatment has not been established. The Canadian product label for eculizumab includes a boxed warning: "Soliris increases the risk of meningococcal infections. Vaccinate patients with a meningococcal vaccine at least 2 weeks prior to 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." During clinical studies, 2 out of 196 vaccinated PNH patients treated with eculizumab experienced a serious meningococcal infection. Prior to beginning eculizumab therapy, all patients and their prescribing physicians are encouraged to enroll 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. SOURCE: Alexion Pharmaceuticals, Inc.
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