eculizumab (Soliris) for PNH

Advice: 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 paroxysmal nocturnal haemoglobinuria (PNH). Evidence of clinical benefit is demonstrated in patients with haemolysis with clinical symptom(s) indicative of high disease activity, regardless of transfusion history.

In a controlled study in patients with transfusion-dependent PNH, eculizumab reduced the rate of haemolysis and improved anaemia compared with placebo. Observational data from a subset of the PNH registry suggest that these benefits may also be achieved in patients with no history of transfusions. Uncontrolled data suggest that eculizumab reduces the incidence of thrombosis in patients with PNH. 

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.