[New Complement Therapeutics in Complement-Related Diseases]

Brain Nerve. 2019 Jun;71(6):555-564. doi: 10.11477/mf.1416201316.
[Article in Japanese]

Abstract

The complement was named after "a complement", a protein molecule that supports an antibody. It was considered previously that the complement mainly participates in protecting against microbial infections. But later, as research on biological functions in complement activation advanced drastically, it was elucidated that the complement could be involved in the onset of various diseases. In 2007, eculizumab (ECZ), an anti-C5 (complement factor 5) monoclonal antibody, was approved as a drug for paroxysmal nocturnal hemoglobinuria (PNH) in the United States. In Japan, ECZ was approved for PNH and atypical hemolytic uremic syndrome (aHUS) in 2010 and 2013, respectively. The success of ECZ created an opportunity for drug companies to develop new therapeutics targeting the complement system; development of complement therapeutics is now a major venture of pharmaceutical companies worldwide. Here, I will provide an outline of the approved complement therapeutics and those that are in development and clinical trial phase currently.

Publication types

  • Review

MeSH terms

  • Antibodies, Monoclonal, Humanized / therapeutic use*
  • Atypical Hemolytic Uremic Syndrome / drug therapy*
  • Clinical Trials as Topic
  • Complement C5 / antagonists & inhibitors
  • Complement Inactivating Agents / therapeutic use*
  • Complement System Proteins*
  • Hemoglobinuria, Paroxysmal / drug therapy*
  • Humans
  • Japan

Substances

  • Antibodies, Monoclonal, Humanized
  • Complement C5
  • Complement Inactivating Agents
  • Complement System Proteins
  • eculizumab