following a full submission assessed under the ultra-orphan process:
eculizumab (Soliris®) is not recommended for use within NHS Scotland.
Indication under review: in adults and children for the treatment of patients with atypical haemolytic uraemic syndrome (aHUS).
Four phase II, open-label, single-arm studies demonstrated the beneficial treatment effect of eculizumab on haematological parameters, renal function and thrombotic microangiopathy events.
The submitting company did not present a sufficiently robust economic analysis and in addition their justification of the treatment’s cost in relation to its health benefits was not sufficient to gain acceptance by SMC.
This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.
Download detailed advice263KB (PDF)
- Medicine name:
- eculizumab (Soliris) aHUS
- SMC ID:
- in adults and children for the treatment of patients with atypical haemolytic uraemic syndrome (aHUS).
- Pharmaceutical company
- Alexion Pharma UK Ltd
- BNF chapter
- Nutrition and blood
- Submission type
- Not recommended
- Date advice published
- 07 February 2016