Alexion Pharmaceuticals, Inc. reported that Soliris (eculizumab), its first-in-class complement inhibitor, has been designated as an orphan drug by the Ministry of Health, Labour and Welfare of Japan. 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 haemoglobinuria (PNH) in Japan.
Soliris was previously granted orphan drug status in the United States and European Union, 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 on December 8, 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.
"The orphan drug designation reflects the therapeutic value of Soliris as a treatment for Japanese patients suffering with PNH, who currently lack an effective drug therapy for their disease," said Dr. Mitsuhiro Omine, Visiting Professor, Showa University Division of Hematology, Internal Medicine, Japan.
"Japanese scientists conducted much of the early research in PNH," said Leonard Bell, M.D., Chief Executive Officer of Alexion. "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."
PNH is a rare blood disorder that strikes people of all ages, with an average age of onset in the early 30s. (1) Approximately 10 percent of all patients first develop symptoms at 21 years of age or younger. (2) PNH develops without warning and can occur in men and women of all races, backgrounds and ages. PNH often goes unrecognized, with delays in diagnosis ranging from one to more than 10 years. (3) The estimated median survival for PNH patients is between 10 and 15 years from the time of diagnosis.
PNH has been identified more commonly among patients with disorders of the bone marrow, including aplastic anaemia (AA) and myelodysplastic syndromes (MDS). In patients with thrombosis of unknown origin, PNH may be an underlying cause.
Prior to approval of Soliris in the U.S. and European Union, there were no therapies specifically available for the treatment of PNH. PNH treatment was limited to symptom management through periodic blood transfusions, non-specific immunosuppressive therapy and, infrequently, bone marrow transplantations -- a procedure that carries considerable mortality risk.
Soliris was approved in March 2007 by the US Food and Drug Administration (FDA) as the first treatment for PNH, a rare, debilitating and life-threatening blood disorder defined by haemolysis, or the destruction of red blood cells. In June 2007, the European Commission (EC) also approved the use of Soliris for the treatment of patients with PNH. Soliris is the first therapy approved in Europe for the treatment of PNH and was the first medicinal product to receive EC approval under the EMEA Accelerated Assessment Procedure.
Alexion Pharmaceuticals, Inc. is a biopharmaceutical company working to develop and deliver life-changing drug therapies for patients with serious and life-threatening medical conditions. Alexion is engaged in the discovery, development and commercialization of therapeutic products aimed at treating patients with a wide array of severe disease states, including haematologic diseases, cancer, and autoimmune disorders.