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    Has all of the relevant evidence been taken into account?

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

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1 Recommendations

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1 Recommendations

1.1

Danicopan is not recommended, within its anticipated marketing authorisation, as an add-on to ravulizumab or eculizumab for treating paroxysmal nocturnal haemoglobinuria (PNH) in adults with residual haemolytic anaemia.

1.2

This recommendation is not intended to affect treatment with danicopan 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 care for PNH with haemolytic anaemia includes the complement component 5 (C5) inhibitors eculizumab and ravulizumab. After a C5 inhibitor, people who still have anaemia (residual haemolytic anaemia) and symptoms of PNH usually have pegcetacoplan.

Evidence from clinical trials shows that danicopan with a C5 inhibitor increases haemoglobin levels and reduces the need for blood transfusions more than a C5 inhibitor alone. There is no direct evidence comparing danicopan with pegcetacoplan and the results from an indirect comparison are uncertain. So it is unclear how well danicopan works compared with pegcetacoplan.

Because of the uncertainty in the clinical evidence and some of the assumptions used to estimate cost effectiveness, the cost-effectiveness estimates for danicopan are also uncertain. More evidence is needed to determine the cost effectiveness of danicopan, so danicopan is not recommended.