NICE rejected efgartigimod outright in 2025, leaving rozanolixizumab as the FcRn class's one still-untested bid for UK access.

An estimated 7,000-10,000 UK patients have generalised myasthenia gravis, with roughly 4,000 having moderate-to-severe disease eligible for novel agents. Eculizumab's (Soliris, AstraZeneca) UK appraisal, TA636, was terminated by NICE in June 2020 after the manufacturer withdrew without submitting cost-effectiveness evidence, leaving refractory patients dependent on Individual Funding Request exceptional-case access only. No cost-per-QALY figure was ever published: this was a manufacturer decision not to pursue UK reimbursement, not a rejection on value grounds. This remains the widest NICE access gap among the UK rare-disease markets in this set: no novel biologic is currently NHS-commissioned for generalised myasthenia gravis.

NICE rejected efgartigimod alfa (Vyvgart/Vyvgart Hytrulo, argenx) for generalised myasthenia gravis in its June 2025 final guidance (TA1069), the first FcRn-antagonist verdict in the class. UK neurology centres continue to access efgartigimod for severe refractory patients via Named Patient Programme, with an estimated 200 UK patients reached this way despite the negative TA. Rozanolixizumab (Rystiggo, UCB) is now the FcRn-antagonist class's one remaining live NICE bid: a positive recommendation with an agreed PAS would be the first transformative access event for the class in the UK, opening NHS commissioning at an estimated 25 specialist neuromuscular centres.

No novel biologic is yet NHS-commissioned for this 4,000-patient eligible population.

Five questions this report answers:

Q1 - What cost-effectiveness evidence would let rozanolixizumab succeed where efgartigimod was rejected in 2025?

Q2 - How is argenx sustaining a 200-patient Named Patient Programme after NICE's 2025 rejection?

Q3 - Which of the ~25 UK neuromuscular centres would first commission rozanolixizumab on a positive verdict?

Q4 - Why was eculizumab's TA636 appraisal withdrawn in 2020 rather than rejected on cost?

Q5 - How many of the UK's 7,000-10,000 gMG patients are eligible for novel agents?

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Mike || Global Pharma Commercial Marketing Head

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