Efgartigimod's June 2025 NICE rejection (TA1069) leaves roughly 4,000 UK generalised MG patients without a recommended novel agent.

Myasthenia gravis is an autoimmune disorder of the neuromuscular junction, most commonly driven by AChR antibodies in 85% of generalised MG, with anti-MuSK and anti-LRP4 subtypes accounting for the remainder. An estimated 7,000-10,000 UK patients have generalised MG; diagnosis is confirmed via AChR-Ab testing at routine NHS immunology labs available at every NHS trust, with anti-MuSK and anti-LRP4 testing reserved for specialist neuromuscular labs. The NHS neuromuscular network comprises approximately 30 specialist centres coordinated through the Muscular Dystrophy UK network, and the Myasthenia Gravis Association UK maintains a patient registry of roughly 3,000 enrolled members.

Eculizumab's own MG appraisal (TA636) was terminated in 2020 after the manufacturer chose not to submit a cost-effectiveness dossier, so NICE never modelled a cost-per-QALY figure for eculizumab in MG. Efgartigimod's appraisal concluded with final guidance TA1069, published 4 June 2025: NICE does not recommend efgartigimod for NHS use, citing gaps and uncertainties in the cost-effectiveness evidence. As a result, an estimated 4,000 UK moderate-to-severe gMG patients remain on the pyridostigmine plus corticosteroid, azathioprine, or mycophenolate backbone, with IVIg or plasma exchange reserved for crises. MGA UK survey data estimate that 30-40% of UK gMG patients have inadequate disease control on current standard therapy.

No novel agent has cleared NICE, leaving inadequate control unresolved for many patients.

Five questions this report answers:

Q1 - What is the size of the UK gMG population with inadequate control after TA1069's rejection?

Q2 - What is the NHS diagnostic pathway for MG, including thymoma screening and thymectomy eligibility?

Q3 - Why was eculizumab's MG appraisal withdrawn, and what does efgartigimod's TA1069 rejection mean next?

Q4 - How many UK patients does the Myasthenia Gravis Association's registry currently enrol?

Q5 - What share of AChR-positive cases account for generalised myasthenia gravis in the UK?

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