Alexion Pharmaceuticals, Inc., a biotechnology company, announced that its wholly-owned subsidiary, Alexion Europe, has submitted a Market Authorization Application (MAA) to the European Medicines Evaluation Agency (EMEA) for Soliris (eculizumab) for the treatment of paroxysmal nocturnal haemoglobinuria (PNH), a rare, life-threatening genetic blood disorder.
If accepted, Alexion anticipates that the evaluation by the EMEA will commence in the fourth quarter. As reported on September 20, 2006, Alexion has previously submitted a Biologics License Application with the US Food and Drug Administration (FDA) for Soliris (eculizumab).
"This MAA submission is the second significant regulatory milestone for Soliris this month, following closely behind our US Biologics License Application submitted to the FDA," said Leonard Bell, M.D., chief executive officer of Alexion. "There currently is no treatment specifically available for PNH, which can be a debilitating disease that often shortens lives. We believe Soliris, if approved, would represent an important treatment option in the management of PNH and in improving the lives of patients diagnosed with the disease. We look forward to working with the EMEA as it reviews the Soliris application."
The MAA submission will be reviewed under the centralized licensing procedure, which, if approval is granted, provides a marketing license valid in all 25 member states of the European Community. The EMEA has determined that the Accelerated Assessment Procedure can be utilized for the Soliris (eculizumab) application review. Accelerated Assessment is given for medicinal products of major therapeutic interest and shortens the timeframe for review by the agency.
The MAA submission includes data from the pivotal phase III TRIUMPH trial, which met all pre-specified primary and secondary endpoints with high levels of statistical significance. Details regarding the TRIUMPH study results are included in an article published in the September 21, 2006 issue of the New England Journal of Medicine. The Soliris (eculizumab) application also includes interim six months data from the open-label Phase III SHEPHERD safety trial. Twelve month data from the SHEPHERD trial is expected to be submitted at a later date to the MAA. The TRIUMPH trial included 87 PNH patients and the SHEPHERD trial enrolled 97 PNH patients. An additional 11 PNH patients were enrolled in an earlier pilot PNH study.
PNH, a rare and life-threatening form of haemolytic anaemia, is an acquired genetic blood disorder characterized by destruction of red blood cells by the body's terminal complement system (a component of the immune system). Patients with PNH lack naturally-occurring complement inhibitors which normally prevent red blood cell destruction. Soliris (eculizumab), a long- acting C5 complement inhibitor, is a humanized monoclonal antibody drug that is designed to selectively block terminal complement activation in the blood of patients with PNH. Soliris (eculizumab) has been granted Orphan Drug Status in the PNH indication from both the FDA and European regulatory agencies. There currently is no approved therapy specifically available for treatment of PNH.
Based upon scientific investigations and presentations of the prevalence of patients diagnosed with abnormal PNH cells in their blood, it is currently estimated that approximately 8,000 - 10,000 people in North America and Europe suffer from PNH. Patients with PNH may suffer from severe haemolysis, anaemia, chronic fatigue, recurrent pain, pulmonary hypertension and intermittent episodes of dark coloured urine, known as haemoglobinuria. Importantly, PNH patients are at increased risk of forming life-threatening blood clots, or thromboses, which are a major cause of death in this disease.