European Commission Approves Strensiq for Treatment of Hypophosphatasia in Pediatric-Onset Patients

Strategic Insights -

Alexion Pharmaceuticals, Inc. has announced that the European Commission has approved Strensiq, a treatment for hypophosphatasia (HPP), a rare and life-threatening metabolic disorder. The approval, announced on September 14, 2015, marks a significant milestone in the treatment of patients with HPP, a disease that affects fewer than 20 patients per million in the general population. The approval is based on clinical data from four pivotal prospective studies and their extensions, comprising 68 patients with pediatric-onset HPP.

Key Takeaways:

  • The European Commission has approved Strensiq, an enzyme replacement therapy, for the treatment of hypophosphatasia (HPP) in pediatric-onset patients.
  • HPP is a genetic and progressive metabolic disease that affects fewer than 20 patients per million in the general population, characterized by defective bone mineralization, muscle weakness, seizures, and respiratory failure leading to premature death.
  • The approval of Strensiq in the EU was based on clinical data from four pivotal prospective studies and their extensions, comprising 68 patients with pediatric-onset HPP.
  • Patients with pediatric-onset HPP treated with Strensiq demonstrated rapid and sustained improvements in bone mineralization, as measured by the Radiographic Global Impression of Change (RGI-C) scale.
  • The most common adverse reactions observed in clinical studies were injection site reactions and injection-associated adverse reactions, which were non-serious and mild to moderate in intensity.
  • Strensiq is the first therapy approved in the European Union (EU) for the treatment of patients with HPP.
  • The EC approval of Strensiq applies to all 28 EU member states as well as Iceland, Norway, and Liechtenstein.

Statistics:

  • 68 patients with pediatric-onset HPP participated in the clinical studies
  • 71% of infant patients treated with Strensiq who required ventilation support remain alive and continue on treatment
  • The natural history of untreated infant HPP patients suggests high mortality, with an overall mortality rate of 73% at 5 years
  • Recurrent and non-healing fractures, profound muscle weakness, debilitating pain, and the requirement for ambulatory assistive devices are long-term clinical sequelae of HPP

Sources:

  • http://www.alexion.com
  • Drug Pipeline: Strensiq
  • Product Information: Strensiq (asfotase alfa)
  • European Commission Approves Strensiq (asfotase alfa) for the Treatment of Hypophosphatasia (HPP) in Pediatric-Onset Patients
  • Alexion Pharmaceuticals, Inc. Announces European Commission Approval of Strensiq (asfotase alfa) for the Treatment of Hypophosphatasia (HPP) in Pediatric-Onset Patients.