Alexion Pharma Canada., a subsidiary of Alexion Pharmaceuticals Inc. (ALXN), announced that Canada's national healthcare regulatory agency, Health Canada, has approved the use of Soliris or eculizumab for the treatment of patients with atypical hemolytic uremic syndrome or atypical HUS to inhibit complement-mediated thrombotic microangiopathy or TMA.
Atypical HUS, or aHUS, is a life-threatening and chronic ultra-rare, genetic disease that progressively damages vital organs, leading to stroke, heart attack, kidney failure and death.
The morbidity and premature mortality in aHUS is caused by chronic uncontrolled activation of the complement system, resulting in the formation of blood clots in small blood vessels throughout the body, known as TMA. Despite historical supportive care, more than half of all patients with aHUS die, require kidney dialysis or have permanent kidney damage within 1 year of diagnosis.
The company said that Health Canada has issued a marketing authorization for adolescents and adults (aged 13-17) with atypical HUS to reduce complement-mediated TMA. Health Canada has also approved Soliris for pediatric patients with atypical HUS under the Notice of Compliance with Conditions (NOC/c) policy based on data collected in a retrospective chart study.
As part of this condition, Alexion has agreed to undertake an additional prospective study in pediatric patients, and to provide additional data from all ongoing and future trials of Soliris in atypical HUS.
Soliris was approved by the U.S. Food and Drug Administration and the European Medicines Agency in 2011 for the treatment of pediatric and adult patients with aHUS to inhibit complement-mediated TMA.
Soliris is also approved in the United States, European Union, Japan and other territories including Canada for the treatment of patients with paroxysmal nocturnal hemoglobinuria, a debilitating, ultra-rare and life-threatening blood disorder.
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