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The content on this page is not current guidance and is only for the purposes of the consultation process.

1 Recommendations

1.1

Efgartigimod is not recommended, within its marketing authorisation, as an add-on to standard treatment for generalised myasthenia gravis in adults who test positive for anti-acetylcholine receptor antibodies.

1.2

This recommendation is not intended to affect treatment with efgartigimod that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS clinician consider it appropriate to stop.

Why the committee made these recommendations

Standard treatment for generalised myasthenia gravis in adults who test positive for anti-acetylcholine receptor antibodies includes surgery, acetylcholinesterase inhibitors or immunosuppressants. Efgartigimod would be used as an add-on to standard treatment.

Clinical trial evidence suggests that efgartigimod plus standard treatment improves symptoms and people's ability to carry out their normal activities compared with standard treatment alone. But it is uncertain if the people in the trial reflect the people who would have efgartigimod in the NHS because the company have proposed a target population with more severe disease.

There are also uncertainties in the economic model that make the likely cost-effectiveness estimates for efgartigimod uncertain. The most likely cost-effectiveness estimates are substantially above what NICE considers an acceptable use of NHS resources. So, efgartigimod is not recommended.