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References

  1. Alexion Pharmaceuticals Inc. Soliris (eculizumab) full prescribing information. United States Food and Drug Administration; 2007 Mar 16.
  2. Hill A, Richards SJ, Hillmen P. Recent developments in the understanding and management of paroxysmal nocturnal haemoglobinuria. Br J Haematol 2007 May;137(3):181-92.
  3. Hillmen P, Lewis SM, Bessler M, Luzzatto L, Dacie JV. Natural history of paroxysmal nocturnal hemoglobinuria. N Engl J Med 1995 Nov 9;333(19):1253-8.
  4. Stahl A, Vaziri-Sani F, Heinen S, et al. Factor H dysfunction in patients with atypical hemolytic uremic syndrome contributes to complement deposition on platelets and their activation. Blood. 2008;111:5307-5315.
  5. Hirt-Minkowski P, Dickenmann M, Schifferli JA. Atypical hemolytic uremic syndrome: update on the complement system and what is new. Nephron Clin Pract. 2010;114:c219-c235.
  6. Clark DA, Butler SA, Braren V, Hartmann RC, Jenkins DE, Jr. The kidneys in paroxysmal nocturnal hemoglobinuria. Blood 1981 Jan;57(1):83-9.
  7. Brodsky R.A. Paroxysmal Nocturnal Hemoglobinuria. In: Hoffman R, Benz E.J.Jr.et al, editors. Hematology: Basic Principles and Practice. 4th ed. Philadelphia, PA: Churchill Livingstone; 2005. p. 419-27.
  8. Nishimura J, Kanakura Y, Ware RE, Shichishima T, Nakakuma H, Ninomiya H, et al. Clinical course and flow cytometric analysis of paroxysmal nocturnal hemoglobinuria in the United States and Japan. Medicine (Baltimore) 2004 May;83(3):193-207.
  9. Rother RP, Rollins SA, Mojcik CF, Brodsky RA, Bell L. Discovery and development of the complement inhibitor eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria. Nat Biotechnol 2007 Nov;25(11):1256-64.
  10. Hillmen P, Muus P, Duhrsen U, Risitano AM, Schubert J, Luzzatto L, et al. Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria. Blood 2007 Dec 1;110(12):4123-8.
  11. Kelly RJ, et al. Long term treatment with eculizumab in paroxysmal nocturnal hemoglobinuria: Sustained efficacy and improved survival. Blood. 2011;117(25):6786-6792.
  12. Hosler GA, Cusumano AM, Hutchins GM. Thrombotic thrombocytopenic purpura and hemolytic uremic syndrome are distinct pathologic entities: a review of 56 autopsy cases. Arch Pathol Lab Med. 2003;127:834-839.
  13. Loirat C, Noris M, Fremeaux-Bacchi V. Complement and the atypical hemolytic uremic syndrome in children. Pediatr Nephrol. 2008;23:1957-1972.
  14. Noris M, Caprioli J, Bresin E, et al. Relative role of genetic complement abnormalities in sporadic and familial aHUS and their impact on clinical phenotype. Clin J Am Soc Nephrol. 2010;5:1844-1859.
  15. Caprioli J, Noris M, Brioschi S, et al; for the International Registry of Recurrent and Familial HUS/TTP. Genetics of HUS: the impact of MCP, CFH, and IF mutations on clinical presentation, response to treatment, and outcome. Blood. 2006;108:1267-1279.
  16. Abstract 1587 entitled "A phase II study of eculizumab in patients with atypical hemolytic uremic syndrome receiving chronic plasma exchange/infusion," presented by Dr. Chantal Loirat at the 16th Congress of the European Hematology Association, Sunday, June 12, 2011.
  17. Abstract 1588 entitled "Eculizumab efficacy and safety in patients with atypical hemolytic uremic syndrome resistant to plasma exchange/infusion," presented by Dr. Chantal Loirat at the 16th Congress of the European Hematology Association, Sunday, June 12, 2011


IMPORTANT SAFETY INFORMATION

The U.S. product label for Soliris includes a boxed warning:

WARNING: SERIOUS MENINGOCOCCAL INFECTIONS

See full prescribing information for complete boxed warning

Life-threatening and fatal meningococcal infections have occurred in patients treated with Soliris. Meningococcal infection may become rapidly life-threatening or fatal if not recognized and treated early (5.1).


  • Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for meningococcal vaccination in patients with complement deficiencies.
  • Immunize patients with a meningococcal vaccine at least 2 weeks prior to administering the first dose of Soliris, unless the risks of delaying Soliris therapy outweigh the risk of developing a meningococcal infection. (See Serious Meningococcal Infections (5.1) for additional guidance on the management of meningococcal infection.)
  • Monitor patients for early signs of meningococcal infections and evaluate immediately if infection is suspected.

Soliris is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS). Under the Soliris REMS, prescribers must enroll in the program (5.2). Enrollment in the Soliris REMS program and additional information are available by telephone: 1-888-soliris (1-888-765-4747).

The most frequently reported adverse reactions in the PNH randomized trial (≥ 10% overall and greater than placebo) are: headache, nasopharyngitis, back pain, and nausea.

The most frequently reported adverse reactions in aHUS single arm prospective trials (≥ 15% combined per patient incidence) are: hypertension, upper respiratory tract infection, diarrhea, headache, anemia, vomiting, nausea, urinary tract infection, and leukopenia.

Please see full Prescribing Information and Important Safety Information for Soliris® (eculizumab).