First treatment launched this week for rare blood disease

摘要

Treatment for patients with a rare blood disorder-paroxysmal nocturnal haemoglobin-uria(PNH)-is now available following the launch of the monoclonal antibody drug eculizumab.Until now PNH patients have received only supportive care.Marketed by Alexion as Soliris and designated as an orphan drug,eculizumab is the first drug to be assessed under the European Medicines Agency's accelerated assessment procedure.