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news.US-based Alexion Pharmaceuticals has reported that Soliris, its first-in-class complement inhibitor, has been designated as an orphan drug by the Ministry of Health, Labour and Welfare of Japan.
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As a result of the designation, a new drug application (NDA) for Soliris would receive priority review from the Japanese regulatory authorities once it is submitted, and the drug would have 10 years of market exclusivity as a treatment for patients with paroxysmal nocturnal hemoglobinuria (PNH) in Japan.
Soliris was previously granted orphan drug status in the US and the EU, and has been in use in the US and European 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 in December 2008 Alexion reported positive results from Aegis.
Alexion expects to include data from the Aegis study in an NDA to be submitted in Japan in 2009. The period of market exclusivity under the orphan drug designation would commence upon approval of that application. The company has begun to establish its commercial organization in Japan in anticipation of a commercial launch of Soliris in that country in 2010.
Leonard Bell, CEO of Alexion, said: “Japanese scientists conducted much of the early research in PNH. In light of the long-standing awareness of PNH in Japan, and compelling results from the Aegis trial, we look forward to the potential to provide the clinical benefits of Soliris to significant numbers of Japanese patients with PNH.”