Japan orphan status for Alexion's Soliris

US firm Alexion Pharmaceuticals reports that Soliris (eculizumab), its first-in-class complement inhibitor, has been designated as an orphan  drug by the Ministry of Health, Labor and Welfare of Japan. As a result  of the designation, a New Drug Application for Soliris would receive  priority review from the Japanese regulatory authorities once it is  submitted, and the product would have 10 years of market exclusivity as  a treatment for patients with paroxysmal nocturnal hemoglobinuria in  that country.

Soliris was previously granted orphan drug status in the USA and  European Union, and has been in use in the these countries following  regulatory approvals in 2007. AEGIS, an open-label registration study  examining Soliris as a treatment for Japanese patients with PNH, was  conducted during 2008 and Alexion has reported positive results from  AEGIS. The firm expects to include data from the AEGIS study in an NDA  to be submitted in Japan in 2009. The company has begun to establish its  commercial organization in Japan in anticipation of a commercial launch  of Soliris in 2010.