On Friday, March 16, 2007, the U.S. Food and Drug Administration (FDA) approved Soliris (eculizumab) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), a disorder in which abnormal breakdown of red blood cells occurs. Made by Alexion Pharmaceuticals, Soliris does not cure the disease, but treats the breakdown of the blood cells. Soliris has proven to be a safe and effective therapy for PNH in three multi-national clinical studies.
Hillmen P, Young NS, Schubert J, Brodsky RA, Socie G, Muus P, Roth A, Szer J, Elebute MO, Nakamura R, Browne P, Risitano AM, Hill A, Schrezenmeier H, Fu CL, Maciejewski J, Rollins SA, Mojcik CF, Rother RP, Luzzatto L
The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. The New England Journal of Medicine 2006 Sep 21;355(12):1233-43.
Hill, A Eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria. Clinical advances in hematology & oncology : H&O 2005 Nov;3(11):849-50.
Hill A, Hillmen P, Richards SJ, Elebute D, Marsh JC, Chan J, Mojcik CF, Rother RP Sustained response and long-term safety of eculizumab in paroxysmal nocturnal hemoglobinuria. Blood 2005 Oct 1;106(7):2559-65. Epub 2005 Jun 28.
Hillmen P, Hall C, Marsh JC, Elebute M, Bombara MP, Petro BE, Cullen MJ, Richards SJ, Rollins SA, Mojcik CF, Rother RP Effect of eculizumab on hemolysis and transfusion requirements in patients with paroxysmal nocturnal hemoglobinuria. The New England Journal of Medicine 2004 Feb 5;350(6):552-9.