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 patients with a body weight of 10kg or above with atypical haemolytic uremic syndrome (aHUS) who are complement inhibitor treatment-naïve or have received eculizumab for at least 3 months and have evidence of response to eculizumab.

SMC restriction: under the advice of the national renal complement therapeutics service

Two single-arm, phase III studies demonstrated the beneficial treatment effect of ravulizumab on complete thrombotic microangiopathy (TMA) response, defined as normalisation of haematological parameters and improvement in renal function.

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.