Alexion Pharmaceuticals, Inc, announced that Canada's national healthcare regulatory agency, Health Canada, has approved the use of Soliris (eculizumab) for the treatment of all patients in Canada with paroxysmal nocturnal hemoglobinuria (PNH), a rare, debilitating and life-threatening blood disorder defined by chronic red blood cell destruction, or haemolysis. Soliris is the first therapy approved in Canada for the treatment of PNH.
"Soliris has had a life-changing impact on patients with PNH and represents the only effective and safe drug therapy available for their disease," said Dr Loree M Larratt, Department of Medicine, Divisional director Clinical Haematology, University of Alberta at Edmonton, and one of the researchers who participated in clinical trials of Soliris. "Haemolysis underlies the significant morbidities and mortality of PNH, and Soliris reduced haemolysis in every patient treated in clinical studies."
Soliris 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 three multi-national clinical studies: Triumph, a placebo-controlled 26-week phase-3 study involving 87 PNH patients, Shepherd, an open-label 52-week phase-3 trial involving 97 PNH patients, and E05-001, a long term extension study. Soliris was approved by the United States 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 US and more than 15 additional countries.
"This approval paves the way for patients with PNH across Canada to begin receiving the clinical benefits of Soliris," said Leonard Bell, chief executive officer of Alexion. "We are currently developing our organization in Canada and we will now work with Canada's public and private healthcare organizations to provide access to Soliris, patient-by-patient, as rapidly as possible."
"We are very excited that Health Canada has been able to move quickly to approve Soliris. We have been hearing from Canadian patients affected by PNH who have been waiting for access to this innovative drug," said Durhane Wong-Rieger, president of the Canadian Organization for Rare Disorders. "We call upon the private, provincial and federal drug plans to act without delay to provide this life-saving therapy to patients as soon as possible."
Soliris was approved in March 2007 by the US Food and Drug Administration (FDA) as the first treatment for PNH, a rare, debilitating and life-threatening blood disorder defined by hemolysis, or the destruction of red blood cells. In June 2007, the European Commission (EC) also approved the use of Soliris for the treatment of patients with PNH.