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    Has all of the relevant evidence been taken into account?

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1 Recommendations

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1 Recommendations

1.1

Zilucoplan 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 zilucoplan 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 healthcare professional 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, corticosteroids and immunosuppressants. For people whose condition does not improve with standard treatment, intravenous immunoglobulin or plasma exchange may also be used. Zilucoplan would be used as an add-on to standard treatment for people who test positive for anti-acetylcholine receptor antibodies and whose condition has not improved with standard treatment alone.

Clinical trial evidence suggests that zilucoplan plus standard treatment improves symptoms and people's ability to carry out their normal activities compared with standard treatment alone. But zilucoplan has not been compared with intravenous immunoglobulin and plasma exchange, so it is unclear how well it works compared with these treatments.

As well as the uncertainties in the clinical evidence, there are uncertainties in the economic model and the cost-effectiveness estimates for zilucoplan. The most likely estimates are substantially above what NICE considers an acceptable use of NHS resources. So, zilucoplan is not recommended.