Zilucoplan has been accepted for restricted use in NHS Scotland as an add-on treatment for eligible adults with refractory anti-AChR antibody-positive generalised myasthenia gravis.

Scotland expands access to zilucoplan

The Scottish Medicines Consortium (SMC) has accepted Zilbrysq (zilucoplan) for restricted use within NHS Scotland for eligible adults with refractory anti-acetylcholine receptor (AChR) antibody-positive generalised myasthenia gravis (gMG).

The decision applies to adults whose disease has not responded adequately to corticosteroids and at least two other treatments, including non-steroidal immunosuppressive therapies and rituximab, or where these treatments are contraindicated or not tolerated.

Patients must also have uncontrolled disease, defined by a Myasthenia Gravis Activities of Daily Living (MG-ADL) score of at least six or a Quantitative Myasthenia Gravis (QMG) score of at least 12, and be considered for or receiving maintenance intravenous immunoglobulin (IVIg) or plasma exchange (PLEX).

According to UCB, the SMC decision makes Scotland the first devolved UK nation to provide routine access to zilucoplan for eligible people with gMG.

Zilucoplan is a complement C5 inhibitor administered by subcutaneous injection. It blocks C5, a component of the immune system involved in the formation of the membrane attack complex, which can contribute to damage at the neuromuscular junction in people with myasthenia gravis.

Phase 3 RAISE data support decision

The SMC decision was supported by results from the Phase 3 RAISE study, a randomised, double-blind, placebo-controlled trial involving adults with anti-AChR antibody-positive generalised myasthenia gravis.

The study found that adding zilucoplan to standard therapy produced statistically significant improvements in MG-ADL scores compared with placebo at Week 12.

Improvements were also reported across secondary measures including QMG and Myasthenia Gravis Composite scores, as well as quality-of-life measures.

The RAISE-XT extension study subsequently provided longer-term data, with improvements reported to be sustained over time.

The SMC also considered evidence from its Patient and Clinician Engagement process. Patients and clinicians highlighted the impact of refractory myasthenia gravis on daily activities, independence and quality of life, alongside the burden associated with hospital-based treatments such as IVIg and PLEX.

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.”

He added that the decision would expand treatment choice for people with gMG and highlighted the option for eligible patients to self-administer zilucoplan at home following appropriate training.

Treatment targets autoimmune muscle weakness

Myasthenia gravis is a chronic autoimmune disease in which antibodies disrupt communication between nerves and muscles, causing fluctuating muscle weakness.

Symptoms can include drooping eyelids, double vision and difficulties with speaking, chewing and swallowing. In severe cases, weakness affecting the muscles involved in breathing can lead to a potentially life-threatening myasthenic crisis.

An estimated 1,900 people in Scotland are living with myasthenia gravis, according to Scottish Government data cited by UCB. Around 80% of people with the condition are estimated to have anti-AChR antibodies.

Charlotte Carpenter, research and partnerships manager at Myaware, said: “Living with refractory myasthenia gravis can affect far more than a person’s physical health.”

She added that unpredictable symptoms can affect daily life, independence, work and family life, and welcomed an additional treatment option for eligible patients in Scotland.

The Medicines and Healthcare products Regulatory Agency (MHRA) granted a marketing authorisation for zilucoplan as an add-on treatment for adults with anti-AChR antibody-positive myasthenia gravis in January 2024.

The SMC decision now establishes restricted routine access through NHS Scotland for the defined group of adults with refractory disease.