Eculizumab, sold under the trade name Soliris among others, is a medication used to treat paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), and neuromyelitis optica. In people with PNH, it reduces both the destruction of red blood cells and need for blood transfusion, but does not appear to affect the risk of death. Eculizumab was the first drug approved for each of its uses, and its approval was granted based on small trials. It is given in a clinic by intravenous (IV) infusion.

Side effects include a risk for meningococcal infections and it is only prescribed to those who have enrolled in and follow a risk evaluation and mitigation strategy, which involves counseling people and ensuring that they are vaccinated. It is a humanized monoclonal antibody functioning as a terminal complement inhibitor.

It has been developed, manufactured, and marketed by Alexion Pharmaceuticals, which had patent exclusivity until 2017. Alexion started selling eculizumab in 2007 making $295 million in 2008 with its stock price rising 130% in 2010. In 2010, it was the most expensive drug in the world. It costs £340,200 (approximately €430,000) per year for ongoing treatment in the UK, $500,000 a year in Canada, and US$409,500 a year in the United States (2010). In 2015, it was the 4th most expensive medication in the US on a per person treated basis

Eculizumab is used to treat atypical hemolytic uremic syndrome (aHUS) and paroxysmal nocturnal hemoglobinuria (PNH). For people with PNH, it improves quality of life and decreases the need for blood transfusions but does not appear to affect the risk of death. It does not appear to change the risk of blood clots, myelodysplastic syndrome, acute myelogenous leukemia, or aplastic anemia. Eculizumab is also used to treat neuromyelitis optica spectrum disorder in adults who are anti-aquaporin-4 (AQP4) antibody positive. Eculizumab is administered in a doctor's office or clinic by intravenous infusion. Women should not become pregnant while taking eculizumab and pregnant women should take it only if it is clearly necessary.

With Regards,
Nancy Ella
Managing Editor
Drug Designing: Open Access