The Scottish Medicines Consortium (SMC) has accepted UCB’s Zilbrysq®▼(zilucoplan) for restricted use within NHS Scotland as an add-on to standard therapy for the treatment of adult patients with refractory anti-acetylcholine receptor (AChR) antibody positive generalised myasthenia gravis (gMG).

The decision applies for the adult patients with refractory anti-AChR antibody positive gMG who(se):

• 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,
• 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,
• are being considered for, or treated with, maintenance intravenous immunoglobulin (IVIg) or plasma exchange.

The SMC’s acceptance makes Scotland the first devolved nation in the United Kingdom to provide routine access to this treatment for eligible patients with MG.

Zilucoplan is the first complement C5 inhibitor to be made routinely available in NHS Scotland for eligible adults. It works by blocking a key part of the immune system that contributes to muscle weakness in people living with MG. By preventing immune-system damage at the connection between nerves and muscles, it can help improve symptoms and daily functioning.

At least 1,900 people in Scotland are living with MG, a rare autoimmune neuromuscular disease that causes fluctuating muscle weakness and fatigue, affecting vision, speech, swallowing, mobility and breathing. Approximately 80% of people with MG are anti-AChR antibody positive.1 Despite the availability of multiple treatment options, some people continue to experience uncontrolled symptoms, resulting in a significant physical, emotional and social burden.

The SMC decision is supported by data from the Phase 3 RAISE study, a randomised, double-blind, placebo-controlled clinical trial. In the study, the addition of zilucoplan to standard therapy resulted in statistically significant improvements in Myasthenia Gravis Activities of Daily Living (MG-ADL) scores compared with placebo. Improvements were also seen across key secondary measures, including Quantitative Myasthenia Gravis (QMG) scores, Myasthenia Gravis Composite (MGC) scores and quality-of-life outcomes. Long-term findings from the RAISE-XT extension study indicated that these improvements were sustained over time.

The SMC also considered evidence presented through its Patient and Clinician Engagement (PACE) process. Participants highlighted the significant burden faced by people living with refractory MG and their caregivers, including reduced quality of life, anxiety associated with symptoms worsening and dependence on hospital-based treatments such as IVIg and PLEX. As a self-administered subcutaneous treatment, zilucoplan may offer greater flexibility for eligible patients by enabling administration at home following appropriate training, potentially reducing the need for travel to specialist treatment centres.

Nico Reynders, UCB UK & Ireland General Manager said: “We welcome the SMC’s decision to make zilucoplan available for eligible patients in Scotland, making Scotland the first devolved nation in the UK to routinely provide access to this treatment. This acceptance marks an important step forward in addressing an unmet need and expanding treatment choice for the MG community. For eligible people living with MG who continue to experience the burden of their condition, access to an innovative targeted treatment with a different mechanism of action offers an important new option. The ability to self-administer zilucoplan at home, following appropriate training, also has the potential to reduce treatment burden and support care delivery closer to home.”

Charlotte Carpenter, Myaware Research and Partnerships Manager, said: “Living with refractory myasthenia gravis can affect far more than a person’s physical health. Unpredictable symptoms can impact daily life, independence, work, family life and overall wellbeing, placing a considerable burden on both patients and their loved ones. At Myaware, we greatly appreciate the steps the SMC have taken to understand and acknowledge this burden. Having access to an additional treatment option in Scotland, particularly one that targets myasthenia, is a positive step forward for the patient community and offers further choice when discussing treatment options with their healthcare team.”