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Avacopan for treating severe active granulomatosis with polyangiitis or microscopic polyangiitis

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1 Recommendations

1.1 Avacopan with a cyclophosphamide or rituximab regimen is not recommended, within its marketing authorisation, for treating severe active granulomatosis with polyangiitis or microscopic polyangiitis in adults.

1.2 This recommendation is not intended to affect treatment with avacopan 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 care for granulomatosis with polyangiitis or microscopic polyangiitis usually starts with corticosteroids plus cyclophosphamide or rituximab.

Clinical trial evidence shows that, after a year, avacopan is more effective at stopping the condition getting worse than standard care. It also suggests fewer side effects from corticosteroids, possibly because of less use overall.

There is uncertainty in the cost-effectiveness model, including:

  • that maintenance treatment does not reflect NHS clinical practice

  • which is the most appropriate estimate for the risk of developing end-stage renal disease.

Taking these uncertainties into account, the most likely cost-effectiveness estimates are above what NICE usually considers an acceptable use of NHS resources. So, avacopan is not recommended.