• Giuseppe Tridente


Eculizumab (Soliris®, Alexion) is an IgG2/4 k monoclonal antibody that binds to the C5 complement factor, thereby inhibiting the formation and activity of the terminal components of complement. The orphan drug status was designated by FDA in 2000 for the treatment of dermatomyositis, in 2001 for idiopathic membranous glomerular nephropathy, in 2003 for paroxysmal nocturnal hemoglobinuria (PNH), in 2009 for atypical hemolytic uremic syndrome (aHUS), and in 2011 for Shiga toxin-induced HUS. EMEA recognized such status in 2003 for the treatment of PNH, in 2009 for aHUS, and in 2012 for infection-associated hemolytic uremic syndrome.


Systemic Inflammatory Response Syndrome Hemolytic Uremic Syndrome Paroxysmal Nocturnal Hemoglobinuria Shiga Toxin Orphan Drug Status 
These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.

Supplementary material

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Supplementary material 1 (XLS 70 kb)


  1. 1.
    Eculizumab (Soliris®) BLA125166 FDA Medical Review, Mar 2007Google Scholar
  2. 2.
    Eculizumab (Soliris®) WC500054208 EMA Summary of product characteristics. Annex I, 2012Google Scholar
  3. 3.
    Eculizumab (Soliris®) WC500119185 EMA Assessment Report, Sept 2011Google Scholar
  4. 4.
    Eculizumab (Soliris®) Product Information, Alexion 2011Google Scholar
  5. 5.
    Kaplan M (2002) Eculizumab (Alexion). Curr Opin Investig Drugs 3:1017–1023PubMedGoogle Scholar
  6. 6.
    Rother RP, Rollins S, Mojcik CF et al (2007) Discovery and development of the complement inhibitor eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria. Nat Biotechnol 25:1256–1264PubMedCrossRefGoogle Scholar
  7. 7.
    Dmytrijuk A, Robie-Suh K, Cohen MH et al (2008) FDA Report: eculizumab (Soliris®9 for the treatment of patients with paroxysmal nocturnal hemoglobinuria. Oncologist 13:993–1000PubMedCrossRefGoogle Scholar
  8. 8.
    Zuber J, Fakhourl F, Roumenlna LT et al (2012) Use of eculizumab for atypical haemolytic uraemic syndrome and C3 glomerulopathies. Nat Rev Nephrol 8:643–657PubMedCrossRefGoogle Scholar
  9. 9.
    Zhou Y, Gong B, Lin F et al (2007) Anti-C5 antibody treatment ameliorates weakness in experimentally acquired Myasthenia Gravis. J Immunol 179:8562–8567PubMedGoogle Scholar
  10. 10.
    Sato D, Callegaro D, Lana-Peixoto et al (2012) Treatment of neuromyelitis optica: an evidence based review. Arq Neuropsiquiatr 70:59–66Google Scholar
  11. 11.
    Eculizumab. WC500054212 EMA Scientific Discussion, 2007Google Scholar
  12. 12.
    Howard J, Barohn R, Freimer M et al (2012) Randomized, double blind, placebo-controlled, crossover, multicenter, phase II study of eculizumab in patients with refractory generalized Myasthenia Gravis (gMG). In: AAN meeting: myasthenia gravis. New Orleans, April 2012, Abstract S35.004Google Scholar
  13. 13.
    Pittock SJ, McKeon A, Mandrekar JN et al (2012) Pilot clinical trial of eculizumab in AQP4-Ig-G-positive NMO. In: AAN meeting, Boston, Oct 2012, Abstract T1826Google Scholar
  14. 14.
    Stegal MD, Chedld MF, Cornell LD (2012) The role of complement in antibody-mediated rejection in kidney transplantation. Nat Rev Nephrol 8:670–678CrossRefGoogle Scholar
  15. 15.
    Smith PK, Shernan SK, Chen JC et al (2011) Effect of C5 complement inhibitor pexelizumab on outcome in high-risk coronary artery bypass grafting: combined results from the PRIMO-CARB I and II trials. J Thorac Cardiovasc Surg 142:89–98PubMedCrossRefGoogle Scholar
  16. 16.
    Testa L, Van Gaal WJ, Bhindi R et al (2008) Pexelizumb in ischemic heart disease: a systematic review and meta-analysis on 15196 patients. J Thorac Cardiovasc Surg 136:884–893PubMedCrossRefGoogle Scholar
  17. 17.
    Noris M, Mescia F, Remuzzi G (2012) STEC-HUS, atypical HUS and TTP are all diseases of complement activation. Nat Rev Nephrol 8:622–633PubMedCrossRefGoogle Scholar
  18. 18.
    Herlitz LC, Bomback AS, Markowitz GS et al (2012) Pathology after eculizumab in dense deposit disease and C3 GN. J Am Soc Nephrol 23: doi: 10.1681/ASN.2011121186
  19. 19.
    Pittock SJ, McKeon A, Mandrekar JN et al (2012) Pilot clinical trial of eculizumab in AQP4-Ig-G-positive NMPO. In: Am Neurol Ass meeting Boston, MA, October, Abstract T1826Google Scholar

Copyright information

© Springer-Verlag Italia 2014

Authors and Affiliations

  1. 1.University of VeronaVeronaItaly

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