Alexion Pharmaceuticals, Inc. announced that the US Food and Drug Administration (FDA) has approved Alexion's Rhode Island manufacturing facility (ARIMF) in Smithfield, Rhode Island as a second source of commercial supply for Soliris (eculizumab). Earlier this year, Alexion reported that the European Medicines Agency had approved ARIMF as a second source of supply for Soliris in the European Union (EU). In addition to sourcing Soliris from ARIMF, the company will maintain its agreement with its long-time contract manufacturer; either source can now meet all of the Company's forecasted commercial and clinical needs for Soliris in the US and EU, which territories accounted for more than 90 per cent of the company's 2009 revenues. Additionally, Alexion has applied for regulatory approvals for ARIMF in other countries where it has, or is establishing, commercial operations.
Soliris is the only drug specifically indicated for the treatment of patients with paroxysmal nocturnal haemoglobinuria (PNH), an ultra-rare, debilitating and life-threatening blood disorder. Soliris was approved by the FDA and the European Commission in 2007, as well as by the regulatory authorities of other countries, including Japan's Ministry of Health, Labour and Welfare (MHLW) in 2010. In addition, more than a dozen clinical studies of Soliris as a treatment for patients with other severe and ultra-rare disorders are currently underway in areas that include nephrology and transplant. Among these are clinical studies with Soliris treatment of two cohorts of patients with atypical haemolytic uremic syndrome (aHUS) as well as transplant patients at elevated risk of antibody-mediated graft rejection.
"Since before the launch of Soliris in 2007, we recognized the critical importance of ensuring the continuity of treatment for patients with ultra-rare and life-threatening diseases," said Stephen P Squinto, executive vice president and head of research and development at Alexion. "Uninterrupted world-wide supply of Soliris provides patients with continued access to stable treatment regimens, and also supports our growing clinical development programmes."
Soliris (eculizumab) is a first-in-class terminal complement inhibitor developed from the laboratory through regulatory approval by Alexion. Soliris has been approved by the healthcare authorities in the US, European Union, Japan and other countries as the first treatment for patients with PNH, an ultra-rare, debilitating and life-threatening blood disorder defined by haemolysis, or the destruction of red blood cells. Prior to these approvals, there was no therapy specifically available for the treatment of PNH.
Patients with PNH in more than 20 countries now have access to Soliris therapy through national or private healthcare providers. As the first terminal complement inhibitor to be approved in countries around the world, Soliris represents a long-sought breakthrough in medical innovation. Alexion's innovative approach to complement inhibition has received some of the pharmaceutical industry's highest honors: the 2008 Prix Galien USA Award for Best Biotechnology Product with broad implications for future biomedical research, and the 2009 Prix Galien France Award in the category of Drugs for Rare Diseases.
Alexion Pharmaceuticals, Inc. is a biopharmaceutical company working to develop and deliver life-changing drug therapies for patients with serious and life-threatening medical conditions.