Alexion drug on fast-track to approval in Japan
Alexion Pharmaceuticals Inc. said Monday its drug Soliris has been declared an orphan drug by Japanese officials, a designation that will help speed the process of getting it approved for patients in the Pacific nation.
Soliris, a drug based on an anti-inflammatory antibody called eculizumab, is Alexion's only commercial product. It is used in the United States and Europe to treat patients with paroxysmal nocturnal hemoglobinuria, or PNH, a rare and potentially fatal genetic blood disorder.
Patients with PNH may suffer from anemia, chronic fatigue and pulmonary hypertension, and are at increased risk of forming life-threatening blood clots. Soliris is the only available treatment targeted specifically for PNH patients.