Health Canada approves Strensiq (asfotase alfa), the first therapy for patients with hypophosphatasia (HPP), an ultra-rare, life-threatening disease

17 August 2015 - Alexion Pharma Canada, a subsidiary of Alexion Pharmaceuticals, Inc., today announced that Health Canada has approved Strensiq (asfotase alfa) as enzyme replacement therapy for patients with confirmed diagnosis of paediatric-onset hypophosphatasia (HPP). Strensiq is the first approved treatment for HPP, an ultra-rare, genetic, metabolic disease in which patients experience devastating, progressive effects on multiple organs of the body, leading to debilitating morbidities or pre-mature mortality.

"Strensiq is critical and pivotal for the treatment of patients with HPP who to date have not had an effective therapy. HPP is a rare and extremely debilitating disease. It has a very high mortality rate in the most severe paediatric cases," said Dr. Cheryl Rockman-Greenberg, Distinguished Professor, Department of Pediatrics and Child Health, University Of Manitoba and Clinician Scientist in the Children's Hospital Research Institute of Manitoba, and lead Canadian investigator in the HPP trials. "In clinical studies to date, Strensiq has greatly reduced mortality in affected infants with HPP with 89 per cent overall survival after three years of treatment compared with almost 100 per cent mortality documented in the past. As well, in clinical studies to date, Strensiq has greatly reduced pain associated with HPP and improved physical functioning of patients, including healing of the 'rickets' and improvement in mobility such as increased speed of walking, jumping, endurance and balance. It is very exciting to now have an approved therapy for patients who previously only faced early death, or had to live with severe disability and pain."

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Michael Wonder

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Michael Wonder