The Pharmaceutical Journal
Vol 279 No 7465 p173
18 August 2007

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First treatment launched this week for rare blood disease

Treatment for patients with a rare blood disorder — paroxysmal nocturnal haemoglobinuria (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. PNH is an acquired disorder that disrupts the formation of blood corpuscles and platelets and affects about 850 patients across England and Wales.

Patients with PNH have a gene that leaves some of their red blood corpuscles abnormally susceptible to break down. Previously, patients have mainly been managed supportively, with folic acid and iron supplements, transfusions, anticoagulants and heparin. Eculizumab prevents the development of a protein that mediates corpuscle destruction in PNH.

Trials have shown that the drug reduces the need for transfusion, cuts thrombosis and increases quality of life. The manufacturers believe that 35–50 per cent of patients could benefit from treatment with eculizumab.