The FDA approved Alexion Pharmaceuticals’ Smithfield, RI, manufacturing facility (ARIMF) as a second source of commercial supply for Soliris®, a drug for paroxysmal nocturnal hemoglobinuria (PNH). The EMEA sanctioned use of ARIMF as a second source of supply for Soliris in the earlier this year.
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 the company’s forecasted commercial and clinical needs for Soliris in the U.S. and EU, which accounted for more than 90% of the company’s 2009 revenues.
Soliris was approved for PNH by the FDA and the European Commission in 2007 as well as by the regulatory authorities of other countries including Japan in 2010. PNH is an ultrarare, debilitating and life-threatening blood disorder.
In addition, more than a dozen clinical studies of Soliris as a treatment for patients with other severe and ultrarare disorders are currently under way 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 as well as transplant patients at elevated risk of antibody-mediated graft rejection.
Alexion licensed the complement-inhibition technology behind Soliris from Oklahoma Medical Research Foundation (OMRF) nearly two decades ago. In 2008, it paid OMRF $10 million to acquire all rights and interests to the patents.