The Food and Drug Administration (FDA) has approved avacopan (Tavneos) as an adjunctive treatment for adult patients with severe active anti-neutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis.

ANCA-associated vasculitis is a systemic disease in which over-activation of the complement pathway further activates neutrophils, leading to inflammation and destruction of small blood vessels. This results in organ damage and failure, with the kidney as the major target, and is fatal if not treated. Currently, treatment for ANCA vasculitis consists of courses of non-specific immunosuppressants (cyclophosphamide or rituximab), combined with the administration of daily glucocorticoids for prolonged periods of time, which can be associated with significant clinical risk including death from infection.

Avacopan is an orally-administered inhibitor of the complement C5a receptor. The approval in ANCA-associated vasculitis was largely based on the results of the pivotal Phase III ADVOCATE trial, published earlier this year in The New England Journal of Medicine (NEJM). The ADVOCATE trial was a global, randomized, double-blind, active-controlled Phase III trial of 330 patients with ANCA-associated vasculitis. Patients were randomized to receive oral avacopan (30 mg twice daily) or oral prednisone on a tapering schedule. All patients received either cyclophosphamide (followed by azathioprine) or rituximab as well.

The primary outcome measure was remission, defined as a Birmingham Vasculitis Activity Score (BVAS) of zero. In this study,  72.3% of patients receiving avacopan and 70.1% receiving prednisone reached remission at week 26 ( P< 0.001 for noninferiority; P=0.24 for superiority). Further, sustained remission at week 52 was observed in 65.7% receiving avacopan and 54.9% receiving prednisone (P< 0.001 for noninferiority; P=0.007 for superiority).

Serious adverse events occurred in 37.3% of the patients receiving avacopan and in 39.0% receiving prednisone.

The most common adverse reactions (≥5% of patients and higher in the avacopan group vs. prednisone group) were: nausea, headache, hypertension, diarrhea, vomiting, rash, fatigue, upper abdominal pain, dizziness, blood creatinine increase, and paresthesia.

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