Alexion Pharmaceuticals Inc. on August 25 announced that the U.S. Food and Drug Administration (FDA) has approved Alexion's Rhode Island manufacturing facility (ARIMF) in Smithfield, R.I., 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 U.S. and the European Union, which territories accounted for more than 90 percent 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 hemoglobinuria (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, Labor 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 hemolytic 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 programs."
