The UK has no NICE-recommended novel agent for myasthenia gravis — efgartigimod's June 2025 rejection (TA1069) leaves the NHS IVIg cost-offset argument as the clearest path to a future resubmission.
Eculizumab (Soliris)'s NICE appraisal for refractory myasthenia gravis (TA636) was terminated in June 2020: the manufacturer, Alexion (now part of AstraZeneca), confirmed it did not intend to submit a cost-effectiveness dossier because the product was not being launched in the UK for this indication, so NICE never modelled a cost-per-QALY figure for eculizumab in MG. Eculizumab reaches UK MG patients only through the Individual Funding Request route on a case-by-case basis, with no NICE-recommended access. This leaves an estimated 4,000 UK moderate-to-severe gMG patients on background immunosuppressive therapy with no NICE-recommended novel agent, a gap the Myasthenia Gravis Association UK has lobbied NICE and NHS England to close.
Efgartigimod (Vyvgart, argenx) was assessed under NICE technology appraisal GID-TA10986 (project ID4003); final guidance TA1069, published 4 June 2025, does not recommend efgartigimod for NHS use, with NICE's committee citing gaps and uncertainties in the cost-effectiveness evidence rather than a specific quantified ICER breach. Its ADAPT trial showed a 68% MG-ADL responder rate, but that clinical benefit did not translate into an NHS-acceptable value case at argenx's submitted price. Patients who started efgartigimod on the NHS before the guidance was published continue under existing funding arrangements; new NHS-funded starts are not currently supported. The NHS IVIg cost offset, an estimated £40–60 million a year spent on immunoglobulin for gMG across roughly 4,000 patients, with supply constrained under the NHS-wide IVIg prioritisation programme (gMG rated Category I), remains the strongest unused lever for a future resubmission if argenx returns with a lower price or a managed access proposal.
UK myasthenia gravis agent NICE and NHS status
| Drug (Brand / INN) | NICE HTA Route | NICE Recommendation & PAS | NHS Access Channel | Cost-Effectiveness Position | Key Payer Risk |
|---|---|---|---|---|---|
| Soliris (eculizumab, MG) | Standard Technology Appraisal — terminated (TA636, 2020) | Appraisal withdrawn; no dossier submitted, no NICE recommendation | Individual Funding Request only (case-by-case) | No NICE cost-effectiveness modelling performed | No NICE precedent set for the anti-complement/FcRn class in MG — access remains IFR-only |
| Vyvgart (efgartigimod alfa) | Standard Technology Appraisal (TA1069, concluded June 2025) | Not recommended — cost-effectiveness evidence judged too uncertain | Pre-guidance NHS starts continue; new NHS-funded starts not supported | No confirmed ICER published; NICE cited evidence gaps rather than a specific cost-per-QALY breach | IVIg cost-offset argument and a lower-price or managed-access resubmission are the main levers for a future positive recommendation |
Sources: NICE TA636 eculizumab MG appraisal (terminated, 2020); NICE TA1069 efgartigimod MG appraisal (not recommended, June 2025); ADAPT trial MG-ADL data; Myasthenia Gravis Association UK access report, 2023; NHS IVIg supply programme Category I/II prioritisation list; NHS England IVIg demand data, 2023.
What this assessment answers
Every section answers a named commercial question your team is asking, scoped to your asset.
Delivers
- NICE efgartigimod MG rejection analysis (TA1069, June 2025 — cost-effectiveness evidence judged too uncertain)
- eculizumab TA636 termination context (no dossier submitted, 2020)
- IFR-only access pathway analysis for both agents
Delivers
- TA1069 committee discussion analysis (evidence gaps cited)
- ADAPT trial MG-ADL responder-rate data (68%)
- SC vs IV administration cost-offset comparison for a future NICE submission
Delivers
- NHS IVIg spend and supply-constraint analysis (Category I prioritisation, £40–60M/year)
- cost-offset modelling methodology
- MGA UK access-gap advocacy context
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Commission This AssessmentWhat's inside
- Why roughly 4,000 UK moderate-to-severe gMG patients have no NICE-recommended novel agent following two separate appraisal outcomes
- How the Myasthenia Gravis Association UK has lobbied NICE and NHS England to close the access gap
- Why Alexion (now part of AstraZeneca) withdrew the eculizumab TA636 appraisal in 2020 without submitting a cost-effectiveness dossier
- How eculizumab access is now limited to case-by-case Individual Funding Requests, with no NICE precedent set for MG
- Why NICE's June 2025 TA1069 verdict cited cost-effectiveness evidence gaps despite a 68% MG-ADL responder rate in the ADAPT trial
- What a lower-price or managed-access resubmission from argenx would need to address to reverse the not-recommended verdict
- Sizing the estimated £40-60 million a year NHS spend on IVIg for gMG under Category I supply prioritisation
- Why the IVIg cost-offset argument is the strongest unused lever for a future efgartigimod resubmission
- How pre-guidance NHS efgartigimod starts continue under existing funding while new NHS-funded starts are not supported
- Mapping the neuromuscular centre network through which Individual Funding Request access to eculizumab is granted
- Why Scotland's SMC and Wales's AWMSG assessments could diverge from NICE's TA1069 not-recommended precedent
- Devolved-nation risk assessment for a future efgartigimod or eculizumab resubmission strategy
Included with every brief
How AXLRx builds this assessment
Prepared by MoatRx analysts.
This assessment is built from NICE's published Technology Appraisal documentation for both eculizumab (TA636, terminated) and efgartigimod (TA1069, not recommended) in myasthenia gravis, the ADAPT trial evidence base, and NHS England's IVIg supply and demand data.
Key sources: NICE TA636 eculizumab MG appraisal (terminated, 2020); NICE TA1069 efgartigimod MG appraisal (not recommended, June 2025); ADAPT trial MG-ADL responder data; argenx UK WAC pricing; Myasthenia Gravis Association UK access report, 2023; NHS IVIg supply programme Category I/II prioritisation list; NHS England IVIg demand data, 2023. Devolved-nation positions (Scotland's SMC, Wales's AWMSG) are assessed separately against NICE's TA1069 precedent.
- NICE eculizumab MG appraisal termination verified against the published NICE TA636 guidance (2020)
- Efgartigimod NICE rejection and committee rationale verified against the published NICE TA1069 guidance (June 2025)
- UK gMG unmet-need population estimate verified against the Myasthenia Gravis Association UK access report, 2023
- NHS IVIg spend and supply-constraint data verified against the NHS IVIg supply programme Category I/II prioritisation list and NHS England IVIg demand data, 2023
Frequently asked questions
Commission this assessment
AXLRx Myasthenia Gravis Payer & HTA is built for market access, HEOR, and pricing teams navigating NICE's June 2025 rejection of efgartigimod (TA1069) and the resubmission pathway for UK myasthenia gravis access. Custom assessment in 72 hours.
Specify indication, payer focus (NICE TA, PAS, IVIg cost offset), and commercial question.
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