avacopan for treating severe active granulomatosis with polyangiitis or microscopic polyangiitis

Last edited 09/2022 and last reviewed 09/2022

Avacopan for treating severe active granulomatosis with polyangiitis or microscopic polyangiitis

Antineutrophil cytoplasmic antibody-associated vasculitis (AAV) is a rare autoimmune disease, with high mortality in the absence of treatment (1)

  • the role of the anaphylatoxin C5a in AAV has been demonstrated in mice
  • C5a is a C5 cleavage product with chemoattracting activity on immune cells through the stimulation of the C5a receptor (C5aR1)
    • targeting C5aR may reduce AAV-induced lesions by inhibiting the recruitment of inflammatory cells to the active site of vasculitis
    • the C5aR1 inhibitor avacopan has been developed for the treatment of AAV
    • avacopan plus rituximab leads to a high rate of AAV remission and allows forgoing of glucocorticoids (1)

NICE state:

  • Avacopan with a cyclophosphamide or rituximab regimen is recommended, within its marketing authorisation, as an option for treating severe active granulomatosis with polyangiitis or microscopic polyangiitis in adults. It is recommended only if the company provides it according to the commercial arrangement

The NICE committee noted that "...Standard care for granulomatosis with polyangiitis or microscopic polyangiitis usually starts with cyclophosphamide or rituximab, followed by maintenance treatment, usually with azathioprine or rituximab. Corticosteroids are also used throughout treatment. Avacopan is an option to be used alongside this standard care...Evidence from a clinical trial shows that, after a year, avacopan with standard care is more effective at stopping the conditions getting worse than standard care alone. It also suggests that using avacopan with standard care results in less toxicity from corticosteroids, possibly because of less use overall..."

Reference:

Related pages:

ANCA - associated vasculitides (AAV)