Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) today announced that the European Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending that the therapeutic indication for Soliris® (eculizumab) be extended to include the treatment of pediatric and adult patients with atypical hemolytic uremic syndrome (aHUS) in Europe. Based on the CHMP's positive recommendation, a final decision from the European Commission is expected in approximately two months, after which the Company will then begin the country-by-country reimbursement processes.
“Importantly, the adopted CHMP opinion recommends to extend marketing authorization for Soliris to treat all patients with aHUS. We look forward to efficiently working with authorities to make Soliris broadly available for European patients suffering with aHUS.”
There are no therapies approved for the treatment of aHUS in Europe. aHUS is an ultra-rare, life-threatening, genetic disease that progressively damages vital organs, leading to stroke, heart attack, kidney failure and death. The morbidity and premature mortality in aHUS is caused by chronic uncontrolled activation of the complement system, resulting in the formation of blood clots in small blood vessels throughout the body, known as thrombotic microangiopathy or TMA. Despite current 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 positive opinion adopted by the CHMP is a significant step towards making Soliris available to children and adults suffering with aHUS in Europe," said Leonard Bell, M.D., Chief Executive Officer of Alexion. "Importantly, the adopted CHMP opinion recommends to extend marketing authorization for Soliris to treat all patients with aHUS. We look forward to efficiently working with authorities to make Soliris broadly available for European patients suffering with aHUS."
The CHMP based its opinion on clinical data from two prospective pivotal Phase 2 open-label clinical trials in adolescent and adult patients with aHUS, and a third retrospective study in children, adolescents, and adults with aHUS. A summary of the CHMP opinion can be accessed at http://www.emea.europa.eu.
Soliris is approved in the US (2007), European Union (2007), Japan (2010) and in other territories, for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH), a debilitating, ultra-rare and life-threatening blood disorder. Alexion has also filed a supplemental Biologics License Application (sBLA) for Soliris as a treatment for patients with aHUS with the U.S. Food and Drug Administration (FDA).
Alexion Pharmaceuticals, Inc.