First UK NICE highly specialized technologies guidance recommends Soliris

England’s health costs watchdog the National Institute for Health and care Excellence (NICE) has today issued final guidance recommending US drugmaker Alexion Pharmaceuticals’ (Nasdaq: ALXN) Solaris (eculizumab) for funding for treating atypical hemolytic uremic syndrome (aHUS).

The guidance, confirming an earlier draft guidance, is the first to be produced as part of NICE’s highly specialized technologies program to evaluate treatments for very rare conditions,NICE noted.

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, noting: “The [Appraisal] 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.”