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  • Question on Consultation

    Has all of the relevant evidence been taken into account?

  • Question on Consultation

    Are the summaries of clinical and cost effectiveness reasonable interpretations of the evidence?

  • Question on Consultation

    Are the recommendations sound and a suitable basis for guidance to the NHS?

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    Are there any aspects of the recommendations that need particular consideration to ensure we avoid unlawful discrimination against any group of people on the grounds of age, disability, gender reassignment, pregnancy and maternity, race, religion or belief, sex or sexual orientation?

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

2

Standard treatment for generalised myasthenia gravis in adults who test positive for anti-acetylcholine receptor antibodies includes surgery to remove the thymus gland , 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 plasma exchange and there is uncertainty in the indirect treatment comparisons between zilucoplan and intravenous immunoglobulin. So it is unclear how well it works compared with these treatments.

As well as the uncertainties in the clinical evidence, there are substantial uncertainties in the economic model. It is not possible to determine a likely cost-effectiveness estimate for zilucoplan. So, zilucoplan is not recommended.