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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

FDA Grants Priority Review for Soliris (eculizumab) BLA for Treatment of Paroxysmal Nocturnal Hemoglobinuria

CHESHIRE, Conn., November 14, 2006 -- Alexion Pharmaceuticals, Inc. today announced that the U.S. Food and Drug Administration (FDA) has accepted for filing the Biologics License Application (BLA) for Soliris (eculizumab) for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH). In addition, the FDA has informed Alexion that it has designated the submission for priority review, which targets an FDA action within six months of the BLA submission date. Alexion submitted the BLA in September 2006. Priority review status is granted by the FDA to products that, if approved, would be a significant improvement over existing therapies.

As previously announced, the European Medicines Evaluation Agency (EMEA) has accepted the Soliris (eculizumab) application for review under the EMEA's Accelerated Assessment Procedure. Review under the Accelerated Assessment Procedure is provided by the EMEA for medicinal products of major therapeutic interest and shortens the timeframe for review by that agency.

"We are extremely pleased that the FDA has accepted the Soliris BLA for filing and designated it for priority review," said Leonard Bell, M.D., Chief Executive Officer of Alexion. "The priority designation supports our belief in the potential of Soliris for the treatment of PNH. There currently is no therapy specifically available for the treatment of PNH, and we believe that Soliris, if approved, would represent an important option in improving the lives of patients with the disease."

The BLA submission is based on data from the pivotal Phase III TRIUMPH trial which met all pre-specified primary and secondary endpoints with 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 BLA submission also includes interim data of at least six months from the open-label Phase III SHEPHERD safety trial. The last patient completed the last visit in the SHEPHERD trial in October 2006 and final 12-month data from the SHEPHERD trial will be submitted to the regulatory applications. 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.

About PNH and Soliris

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 an investigational humanized 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: November 2006

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