NICE backs UCB drug as first uncontrolled gMG therapy

UCB's Rystiggo can be used by the NHS as a backup treatment for people with the rare autoimmune and neuromuscular disease generalised myasthenia gravis who don't respond well enough to standard therapies.

New guidance from reimbursement authority NICE has recommended Rystiggo (rozanolixizumab) for people with gMG who test positive for anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibodies and who still have poorly controlled symptoms, even after trying standard treatments.

It is the first targeted treatment for gMG to be cleared for NHS use in England, after two others – Argenx's Vyvgart (efgartigimod) and UCB's Zilbrysq (zilucoplan) – were deemed not to be cost-effective in previous guidance.

NICE's Zilbrysq decision was appealed – a process that is still playing out – but has already resulted in changes to the appraisal parameters that have paved the way for Rystiggo's approval, according to patient advocacy organisation Muscular Dystrophy UK. Like muscular dystrophy, gMG attacks neuromuscular junctions, leading to muscle weakness and extreme tiredness, and can affect breathing, speech, swallowing, eyesight, and movement.

The appeal ruled that it was unreasonable to exclude minimal symptom expression (MSE) - a point where someone has no or very few symptoms – from NICE cost-effectiveness modelling. The charity said patient feedback indicated MSE is "truly life changing" and should be considered in NICE decision-making.

In a statement, NICE said interim funding for Rystiggo will be made available straight away via the Innovative Medicines Fund (IMF), designed to accelerate access to novel, non-cancer medicines while additional data on benefits are gathered. It estimates that around 800 adults in England could benefit from the new treatment.

UCB's drug can be self-administered at home via a short subcutaneous infusion, after training by a healthcare professional, and means that people living with gMG can avoid having to attend hospital for several days of treatments, such as IV immunoglobulin (IVIg) or plasma exchange (PLEX), which until now has been the only option for those whose conditioned cannot be managed effectively with steroids and immunosuppressant medicines.

Wales and Northern Ireland usually follow NICE recommendations, while Scotland's SMC authority carries out its own appraisals. The SMC turned down Rystiggo in January 2025, and it's not clear at the moment whether the drug will be re-appraised following NICE's decision.

The chief executive of myasthenia charity Myaware, Marc Smith, thanked the tireless efforts of gMG community patients "who have supported us throughout the appraisals of many new medicines."

He added: "Without our members, we would be unable to authentically capture the reality of living with gMG effectively enough to convince decision makers of this unmet need."

Rystiggo is a key growth product for UCB, with sales rising 71% last year to reach €332 million ($389 million).