Soliris

Soliris is a medicine used to treat adults and children with paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS). These are life-threatening genetic diseases that cause the breakdown of red blood cells resulting in various medical complications. PNH results in anaemia (low red blood cell counts), thrombosis (blood clots in the blood vessels), pancytopenia (low counts of blood cells) and dark urine, while aHUS results in anaemia, thrombocytopenia (a decrease in the number of platelets, components that help the blood to clot) and kidney failure. Soliris is used to treat adults and children aged 6 years and above with myasthenia gravis (a disease where the immune system attacks and damages muscle cells causing muscle weakness), in whom other medicines do not work (refractory generalised myasthenia gravis, refractory gMG) and who have a specific antibody in their body called AChR antibody. Soliris is also used to treat adults with neuromyelitis optica spectrum disorder (NMOSD), a disease where the immune system damages nerve cells causing problems mostly with the optic (eye) nerve and the spinal cord (nerve tissue that runs from the base of the skull down the center of the back). It is used in patients who have an antibody called AQP4 and whose disease is relapsing (where the patient has attacks \[relapses\] between periods with no symptoms). Soliris contains the active substance eculizumab. These diseases are rare, and Soliris was designated an ‘orphan medicine’ (a medicine used in rare diseases). Further information on the orphan designations can be found on the European Medicines Agency’s website ( [PNH](/en/medicines/human/orphan-designations/eu-3-03-166): 17 October 2003; [aHUS](/en/medicines/human/orphan-designations/eu-3-09-653): 24 July 2009; [myasthenia gravis](/en/medicines/human/orphan-designations/eu-3-14-1304): 29 July 2014; [NMOSD](/en/medicines/human/orphan-designations/eu-3-13-1185): 24 April 2019).