NICE has today issued final guidance recommending eculizumab (Soliris, Alexion) for funding for treating atypical Haemolytic Uraemic Syndrome (aHUS). The guidance is the first to be produced as part of NICE’s highly specialised technologies programme to evaluate treatments for very rare conditions.

aHUS is a life-threatening disease affecting around 200 people in England, with 20–30 new patients diagnosed with the condition each year. It causes inflammation of blood vessels and the formation of blood clots throughout the body. People with aHUS are at constant risk of sudden and progressive damage to, and failure of vital organs, particularly the kidneys.

 “aHUS is a very distressing condition that imposes a significant burden both on those with the condition and their carers and families. We are therefore pleased to be able to recommend eculizumab for funding” said NICE Chief Executive Sir Andrew Dillon.

“The Committee accepted that eculizumab is a step change in the management of aHUS and can be considered a significant innovation for a disease with a high unmet clinical need. It offers people with aHUS the possibility of avoiding end-stage renal failure, dialysis and kidney transplantation, as well as other organ damage.

For more details, go to: https://www.nice.org.uk/news/press-and-media/first-nice-highly-specialised-technologies-guidance-recommends-eculizumab-soliris-for-treating-very-rare-life-threatening-blood-disorder