Avacopan, a complement inhibitor approved for ANCA-associated vasculitis, has been under a cloud since its pivotal trial was retracted after a data re-adjudication. The FDA has asked the manufacturer to withdraw it, and European regulators revoked its authorization. In this context, the new Italian registry study offers independent real-world evidence.
Researchers identified 46 patients who started avacopan between 2022 and 2024 across 18 centers. Among 32 followed for at least a year, 31 achieved clinical remission, defined as a Birmingham Vasculitis Activity Score of zero. Median estimated glomerular filtration rate rose from 30.5 to 53.5 mL/min/1.73 m² at week 52, and mean daily prednisone-equivalent doses fell from 23 mg to 5 mg by week 12. Fifty-five percent of patients stopped corticosteroids entirely.
The study has important limitations: it is small, uncontrolled, and does not resolve safety questions. Two patients had grade 1 liver enzyme elevations, and nine patients discontinued treatment, including four for infections. One patient who relapsed died from a fungal infection after receiving rituximab. Patients with relapsing disease were less likely to achieve steroid-free remission than newly diagnosed patients (40% vs 75%). The authors argue that prospective real-world studies are essential to assess effectiveness and safety in unselected populations.