EU approves Strensiq (asfotase alfa) to treat pediatric-onset hypophosphatasia (HPP)- Alexion Pharmaceuticals Inc.

Alexion Pharmaceuticals, Inc. announced that the European Commission has approved Strensiq (asfotase alfa) for long-term enzyme replacement therapy in patients with paediatric-onset hypophosphatasia (HPP) to treat the bone manifestations of the disease. The Summary of Product Characteristics 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.

Comment: Asfotase alfa is designed to address the underlying cause of HPP by normalizing the genetically defective metabolic process, and preventing or reversing the severe and life-threatening complications of life-long dysregulated mineral metabolism.