Alexion Pharmaceuticals has submitted a new drug application for Soliris as a treatment for patients with paroxysmal nocturnal hemoglobinuria to Japan’s Pharmaceuticals and Medical Devices Agency.
The new drug application (NDA) included data from Aegis, an open-label registration study examining Soliris as a treatment for Japanese patients with paroxysmal nocturnal hemoglobinuria (PNH).
Alexion CEO Leonard Bell said that the submission could be an important step toward improving the lives of patients suffering from PNH in Japan.
“Given Japan’s long-standing leadership in PNH research, the debilitating nature of this rare and serious disease, and the markedly positive results of the Aegis study, we know that the Japanese medical community is eager to provide Soliris to patients as quickly as the regulatory process can allow,” Bell said.
In January 2009, Soliris was designated as an orphan drug by Japan’s Ministry of Health, Labour and Welfare. As a result of the designation, the NDA will receive priority review from the Pharmaceuticals and Medical Devices Agency, and the drug would have ten years of market exclusivity as a treatment for patients with PNH in Japan upon approval.
Alexion has begun to establish its commercial organisation in Japan in anticipation of a commercial launch of Soliris in that country in 2010.