ravulizumab (Ultomiris)

following a full submission under the orphan equivalent medicine process:

ravulizumab (Ultomiris®) is accepted for restricted use within NHSScotland.

Indication under review: for the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH):
• In patients with haemolysis with clinical symptom(s) indicative of high disease activity
• In patients who are clinically stable after having been treated with eculizumab for at least the past 6 months.

SMC restriction: under the advice of the national PNH service.

In two open-label, randomised, phase III studies, ravulizumab was non-inferior to another complement inhibitor across a range of relevant outcomes assessing the control of haemolysis.

This advice applies only in the context of an approved NHSScotland Patient Access Scheme (PAS) arrangement delivering the cost-effectiveness results upon which the decision was based, or a PAS/ list price that is equivalent or lower.

This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.