People with a rare blood disorder that can lead to fatal kidney failure will soon receive treatment on the NHS.

The National Institute for Health and Care Excellence (NICE) has recommended the drug eculizumab that will help some 200 people in England who suffer from the disorder each year.

Produced by Alexion, eculizumab, treats atypical Haemolytic Uraemic Syndrome (aHUS), an extremely rare but life-threatening disease which 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.

Eculizumab is being evaluated as part of a new programme at NICE that looks at highly specialised technologies which may benefit people with very rare diseases at a high price.

NICE’s estimates that this drug will cost the NHS up to £58 million in the first year, rising to £82 million after 5 years. 

As well as having a significant negative impact on quality of life, the prognosis for people with aHUS is poor, with around 10% to 15% of people with the disease dying in the initial, acute phase. The majority of people with aHUS – up to 70% – develop end stage kidney failure requiring dialysis. One patient in 5 has aHUS affecting organs other than the kidneys, most commonly the brain or heart.

In around 70% of patients, aHUS is associated with an underlying genetic or acquired abnormality of proteins in the immune system. Eculizumab inhibits the disease process by blocking pro-thrombotic and pro-inflammatory processes which in aHUS can lead to cellular damage in small blood vessels throughout the body, renal failure and damage to other organs.