A NEW retrospective study from Japan has provided real-world insights into the efficacy and safety of avacopan, an oral C5a receptor antagonist, for antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV). The findings confirm avacopan’s potential as a glucocorticoid-sparing therapy, though concerns remain about its side effect profile and high rate of early discontinuation.
Conducted at a single medical center, the study evaluated 21 patients with newly diagnosed or relapsed AAV who received avacopan. Clinical remission rates were high, with 95.2% achieving remission at six months and 90.4% maintaining remission at 12 months. These results align with data from the phase 3 ADVOCATE trial, which previously demonstrated avacopan’s effectiveness as an alternative to traditional glucocorticoid therapy.
However, adverse events were common, occurring in nearly half (47.6%) of patients. Elevated liver enzyme levels were the most frequent complication, affecting 38.1% of participants. As a result, nine patients (42.9%) discontinued avacopan early. Despite this, these patients still achieved remission and required lower glucocorticoid doses, indicating avacopan’s impact on disease control even after stopping the drug.
The study highlights both the potential benefits and challenges of avacopan in real-world clinical settings. While it effectively induces and sustains remission, its safety profile requires further examination. The authors emphasise the need for additional research to optimise treatment strategies and mitigate side effects, particularly liver toxicity.
Aleksandra Zurowska, EMJ
Reference
Tagami G et al. Efficacy and safety of avacopan in antineutrophil cytoplasmic autoantibody-associated vasculitis: a retrospective cohort study in Japan. BMC Rheumatol. 2025;DOI: 10.1186/s41927-025-00456-4.
