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Soliris

Generic name: eculizumab
Treatment for: Paroxysmal Nocturnal Hemoglobinuria, Hemolytic Uremic Syndrome, Myasthenia Gravis

Update: Soliris Now FDA Approved - March 16, 2007

Alexion Pharmaceuticals Submits Biologics License Application for Soliris (eculizumab)

CHESHIRE, Conn., September 20, 2006 -- Alexion Pharmaceuticals, Inc. today announced that it has submitted a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) for its lead product candidate Soliris (eculizumab) as a treatment for patients diagnosed with paroxysmal nocturnal hemoglobinuria (PNH), a rare life-threatening genetic blood disorder.

"There currently is no treatment specifically available for PNH, which can be a debilitating disease that often shortens lives," said Leonard Bell, M.D., Chief Executive Officer of Alexion. "We believe Soliris represents a significant advance in the management of PNH and in improving the lives of patients diagnosed with the disease. We look forward to working with the FDA as it reviews our application and we remain on track to submit a Marketing Authorization Application (MAA) in Europe by the end of this year."

The BLA submission is based on 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 today in the New England Journal of Medicine. The BLA submission also includes interim data of at least six months from the open-label Phase III SHEPHERD safety trial. Final 12-month data from the SHEPHERD trial will be submitted to the application. The TRIUMPH trial included 87 PNH patients and the SHEPHERD trial has enrolled 97 PNH patients. An additional 11 PNH patients were enrolled in an earlier pilot study.

Alexion has requested Priority Review designation for this BLA from the FDA. If granted, the FDA has up to six months from submission date to take action on the BLA. Priority Review may be granted to drug products that provide significant improvement compared to existing treatments for a disease. Alexion has already been granted Accelerated Assessment by the EMEA for Soliris (eculizumab) in Europe once the MAA is submitted.

PNH, a rare and life-threatening form of hemolytic anemia, is an acquired genetic blood disorder characterized by destruction of red blood cells by the body's 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 terminal complement inhibitor, is a monoclonal antibody drug that is designed to selectively block terminal complement activation and thereby restore complement inhibition 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 to 10,000 people in North America and Europe suffer from PNH. Patients with PNH may suffer from severe hemolysis, anemia, chronic fatigue, recurrent pain, pulmonary hypertension and intermittent episodes of dark colored urine, known as hemoglobinuria. 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.

Posted: September 2006

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