Background/Purpose: Avacopan, a selective C5aR1 inhibitor, has demonstrated efficacy and safety over 52 weeks in patients with anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis. However, efficacy and safety data on avacopan beyond 52 weeks are limited. Here, we describe the experience with avacopan beyond 52 weeks from the EAP.

Methods: Safety data in patients with severe, active granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA) within the EAP were recorded in a global safety database from Feb 2019 – Apr 2023. Adverse events (AE) included a lack of effect and other events (i.e., relapse or worsening of disease).

Results: A total of 19 patients were treated with avacopan beyond 52 weeks within the EAP. Average age was 47 years, with 13 patients (68%) diagnosed with GPA and 6 (32%) with MPA. The median duration of therapy was 17 months (range 12–45). A total of 9 AEs were recorded in 2 patients (10.6%) (Table 1). One vasculitis flare was recorded 6 months after avacopan initiation and coincided with an unintended dose reduction to 20 mg BID, due to a product supply issue during COVID. The event was well-managed with rituximab, with no additional use of glucocorticoids, and avacopan 30 mg BID was reinstated. No further cases of a lack of effect, worsening of disease, or disease relapse were reported. Data regarding concomitant medications did not indicate a decline in the patients’ status during treatment. No treatment discontinuations due to AEs were recorded.

Conclusion: These results suggest that continuation of avacopan beyond 52 weeks is generally well-tolerated in patients with GPA and MPA and may be effective in terms of disease control. Limitations of this program include low patient number, potential underreporting, and incomplete data.

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ACR Meeting Abstracts - https://acrabstracts.org/abstract/efficacy-and-safety-experience-with-avacopan-beyond-52-weeks-in-the-early-access-program-eap/