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 in the U.S. and EU in 2007, as well as by the regulatory authorities of other countries, including Japan's Ministry of Health, Labour and Welfare (MHLW) in 2010. Also, more than a dozen clinical studies of Soliris as a treatment for other severe and rare disorders are currently underway in areas that include nephrology and transplant.
“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 R&D 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.”