1 September 2015 - Alexion Pharmaceuticals, Inc. announced today that the European Commission has approved Strensiq (asfotase alfa) for long-term enzyme replacement therapy in patients with pediatric-onset hypophosphatasia (HPP) to treat the bone manifestations of the disease. The Summary of Product Characteristics (SmPC) states that HPP is associated with multiple bone manifestations including rickets/osteomalacia, altered calcium and phosphate metabolism, impaired growth and mobility, respiratory compromise that may require ventilation, and vitamin B6-responsive seizures. Strensiq is the first therapy approved in the European Union (EU) for the treatment of patients with HPP, a life-threatening, ultra-rare metabolic disorder. Alexion expects to begin serving patients in Germany in October and is now commencing reimbursement processes with healthcare authorities in each of the major European countries.

“Hypophosphatasia is an extremely rare disorder that can have devastating consequences for patients and families. Without treatment, patients may face significant challenges related to development, growth, and mobility, with an extremely high risk of mortality in infants,” said PD Dr. med Christine Hofmann, Children’s Hospital, University of Würzburg, Pediatric Rheumatology and Osteology Section, Würzburg, Germany. “I am very pleased that patients with pediatric-onset HPP in Europe now have an approved treatment that addresses the underlying cause of their genetic, lifelong metabolic disease by replacing tissue non-specific alkaline phosphatase.”

For more details, go to: http://news.alexionpharma.com/press-release/company-news/european-commission-grants-marketing-authorization-strensiq-asfotase-alfa