zilucoplan (Zilbrysq)

following a resubmission assessed under the orphan equivalent medicine process:

zilucoplan (Zilbrysq®) is accepted for restricted use within NHSScotland.

Indication under review: as an add-on to standard therapy for the treatment of generalised myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive.

SMC restriction: for adult patients with refractory anti-AChR antibody positive gMG if:

• the disease has not responded adequately to corticosteroids and at least two other treatments including non-steroidal immunosuppressive therapies (NSISTs) and rituximab, or these options are contraindicated or not tolerated, and
• the disease is uncontrolled, as defined by a Myasthenia Gravis-Activities of Daily Living (MG-ADL) score of six or greater or a Quantitative Myasthenia Gravis (QMG) score of 12 or greater, and
• patients are being considered for, or treated with, maintenance intravenous immunoglobulin (IVIg) or plasma exchange (PLEX).

In a double-blind phase III study, the addition of zilucoplan to standard therapy was associated with a statistically significant reduction in patient-reported myasthenia gravis symptoms and their effects on daily activities compared with placebo.

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.