What is "eculizumab"

n. A monoclonal antibody directed against the complement protein C5.

Eculizumab ( INN and USAN; trade name Soliris) is a humanized monoclonal antibody that is a terminal complement inhibitor. In people with paroxysmal nocturnal hemoglobinuria (PNH) it improves quality of life but does not appear to affect the risk of death. Its safety is unclear as of 2014. It is the first approved therapy for paroxysmal nocturnal hemoglobinuria. Eculizumab is also the first agent approved treatment of atypical hemolytic uremic syndrome (aHUS) with likely benefit based on two small trials.

Eculizumab was developed and is manufactured and marketed by Connecticut-based Alexion Pharmaceuticals. It was approved by the United States Food and Drug Administration (FDA) on March 16, 2007 for the treatment of PNH, and on September 23, 2011 for the treatment of aHUS. It was approved by the European Medicines Agency for the treatment of PNH on June 20, 2007, and on November 24, 2011 for the treatment of aHUS. Eculizumab is currently being investigated as a potential treatment for other rare disorders. Eculizumab has exclusivity rights until 2017 which protects it from competition from biosimilar applications until 2017.

In 2010 Soliris was the most expensive drug in the world. It costs £340,200 (approximately €430,000) per year for ongoing treatment in the UK and $500,000 a year in Canada. and US$409,500 a year in the United States (2010). In the case of the rarest diseases that afflict fewer than 10,000 people, biotech companies who own the only approved drugs to treat those diseases "can charge pretty much whatever they want." "Before testing Soliris for PNH, Alexion tested the drug for rheumatoid arthritis, which afflicts 1 million Americans. The trials failed. But if it had worked for arthritis, Alexion would likely have had to charge a much lower price for this use, as it would have to compete against drugs that cost a mere $20,000." Alexion started selling Soliris in 2008 making $295 million in 2007 with its stock price rising 130% in 2010.