1.1 Ravulizumab is recommended, within its marketing authorisation, as an option for treating atypical haemolytic uraemic syndrome (aHUS) in people weighing 10 kg or more:
who have not had a complement inhibitor before or
whose disease has responded to at least 3 months of eculizumab treatment.
It is recommended only if the company provides ravulizumab according to the commercial arrangement (see section 2).
Why the committee made these recommendations
Current treatment for aHUS is eculizumab infusions every 2 weeks. People would have ravulizumab infusions every 8 weeks.
Clinical trial evidence suggests that ravulizumab is effective for treating aHUS. But ravulizumab has not been compared directly with eculizumab. The results of indirect comparisons are uncertain, but it is likely that ravulizumab and eculizumab are equally effective because they work in a similar way. Because people have ravulizumab less often than eculizumab it improves quality of life.
Ravulizumab costs less than eculizumab and the cost-effectiveness estimates are within what NICE normally considers an acceptable use of NHS resources. So, ravulizumab is recommended.