. 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.

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.(1)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.(2,3) 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.(4)

"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).

About aHUS

aHUS is a chronic, ultra-rare, and life-threatening disease in which a genetic deficiency in one or more complement regulatory genes causes life-long uncontrolled complement activation, resulting in complement-mediated thrombotic microangiopathy (TMA), the formation of blood clots in small blood vessels throughout the body.(1,2) Permanent, uncontrolled complement activation in aHUS causes a life-long risk for TMA, which leads to sudden, catastrophic, and life-threatening damage to the kidney, brain, heart, and other vital organs, and premature death.(2,3) More than half of all patients with aHUS die, require kidney dialysis or have permanent kidney damage within 1 year of diagnosis.(4) Patients with aHUS who receive a kidney transplant commonly experience subsequent systemic TMA, resulting in a 90% transplant failure rate.(5)

aHUS affects both children and adults. In a large group of aHUS patients, 60% were first diagnosed at younger than 18 years of age.(6) Complement-mediated TMA also causes reduction in platelet count (thrombocytopenia) and red blood cell destruction (hemolysis). While mutations have been identified in at least ten different complement regulatory genes, mutations are not identified in 30-50% of patients with a confirmed diagnosis of aHUS.(6)

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