Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) today announced that the European Commission (EC) has extended the therapeutic indication for Soliris® (eculizumab) to include the treatment of pediatric and adult patients with atypical hemolytic uremic syndrome (aHUS). Soliris is the first therapy approved in the European Union for the treatment of aHUS, an ultra-rare, life-threatening, chronic, genetic disease that progressively damages vital organs, leading to stroke, heart attack, kidney failure and death.
“The EC approval marks another milestone for Soliris and brings life-transforming hope another step closer to families in Europe living with this severe, devastating and life-threatening disease”
The morbidity and premature mortality in aHUS are caused by chronic uncontrolled activation of the complement system, resulting in the formation of multiple blood clots in small blood vessels throughout the body, known as thrombotic microangiopathy or TMA. Despite historical supportive care, more than half of all patients with aHUS die, require kidney dialysis or have permanent kidney damage within 1 year of diagnosis.
"The approval of Soliris for aHUS in Europe is a major milestone for patients with aHUS, whose uncontrolled complement activation leads to progressive organ failure and a broad range of life-threatening outcomes," said Christophe Legendre, M.D., Professor of Nephrology at University Rene Descartes-Hôpital Necker in Paris. "In clinical studies, chronic treatment with Soliris resulted in a rapid and sustained reduction in complement-mediated TMA. This drug alters the course of aHUS and can make a dramatic difference in patients' lives."
Soliris, a first-in-class terminal complement inhibitor, specifically targets uncontrolled complement activation. The EC has granted marketing authorization for Soliris to treat pediatric and adult patients with aHUS. The Clinical Particulars section of the EU label states, "Soliris treatment is recommended to continue for the patient's lifetime, unless the discontinuation of Soliris is clinically indicated," as described in the Special warnings and precautions for use subsection. Alexion will begin reimbursement discussions with healthcare authorities in major European countries, and expects to start serving patients with aHUS in initial major European countries in the first half of 2012, with additional major European countries commencing through mid-2013.
"The EC approval marks another milestone for Soliris and brings life-transforming hope another step closer to families in Europe living with this severe, devastating and life-threatening disease," said Leonard Bell, M.D., Chief Executive Officer of Alexion. "We will work diligently with the healthcare authorities in individual countries to make Soliris available to children and adults with aHUS as quickly as possible."
Soliris was previously approved by the U.S. Food and Drug Administration (FDA) on September 23, 2011 for the treatment of patients with aHUS to inhibit complement-mediated TMA. Soliris is also approved in the United States, European Union, Japan and other territories for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH), a debilitating, ultra-rare and life-threatening blood disorder.