NICE's eculizumab appraisal was terminated before any cost-effectiveness review ran, leaving 2,000 to 3,000 UK gMG patients without a commissioned biologic.

Eculizumab (Soliris, AstraZeneca) is NICE's clearest cautionary precedent in rare disease: despite FDA and MHRA approval for gMG, the NICE appraisal, TA636, published 30 June 2020, was terminated before a cost-effectiveness review ever took place, because AstraZeneca never submitted the evidence NICE required. No ICER was modelled or published for the drug in this indication. The result is a UK gMG market with zero NICE-commissioned novel biologic: refractory patients rely on IVIg maintenance and plasma exchange, with only case-by-case NHS Individual Funding Request access to eculizumab for the highest-risk 50-100 patients a year, an expensive, administratively heavy route NHS commissioning managers are motivated to retire.

Efgartigimod (Vyvgart Hytrulo, argenx), the first FcRn antagonist, cleared MHRA approval but NICE published its final guidance, TA1069, on 4 June 2025 declining to recommend it for NHS commissioning. No FcRn antagonist has yet cleared NICE's price bar in gMG. Of an estimated 12,000-15,000 UK gMG patients, 2,000-3,000 are refractory to immunosuppressant therapy and represent significant pent-up demand, a population that has now watched two consecutive gMG biologics fail to secure NHS commissioning. The IVIg backbone these patients currently receive costs the NHS an estimated £1.5-5 million a year in gMG alone, adding institutional motivation toward a novel-biologic alternative that can clear NICE's threshold.

Two consecutive gMG biologics have now failed to secure NHS commissioning.

Five questions this report answers:

Q1 - What does NICE's terminated eculizumab appraisal mean for a new gMG biologic's pricing?

Q2 - How large is the UK refractory gMG population with no NICE-commissioned biologic option?

Q3 - What cost-offset arguments make a UK gMG NICE submission viable?

Q4 - What deliverable formats come with a commissioned UK gMG launch readiness assessment?

Q5 - What must a pre-launch myasthenia gravis asset prove to succeed in the UK?

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Live report page:  https://axlrx.ai/myasthenia-gravis/uk/launch-readiness/

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Mike || Global Pharma Commercial Marketing Head

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