Avacopan May Be Safe, Efficacious, and Superior to Other Treatments in ANCA-Associated Vasculitis

GCA on persons hand
Researchers evaluated the efficacy and safety of avacopan for the treatment of ANCA-associated vasculitis.

The following article is a part of conference coverage from the European League Against Rheumatism (EULAR) 2020 E-Congress, held online from June 3 to 6, 2020. The team at Rheumatology Advisor will be reporting on the latest news and research conducted by leading experts in rheumatology. Check back for more from the EULAR 2020 E-Congress.


Avacopan may be safe and efficacious, and yields benefits not seen with other treatments, including chronic prednisone therapy, in anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV), according to study results presented at the European League Against Rheumatism (EULAR) E-Congress, held online from June 3 to 6, 2020.

In a phase 3 double-blind study, 330 patients were randomly assigned to receive prednisone (n=164) or avacopan (n=166) in combination with other treatments during a period of 52 weeks. Patients were stratified based on treatment regimen, ANCA serotype, and diagnosis type (new or relapse). Efficacy end points were disease remission at week 26 and sustained remission at week 52. For the purposes of the study, disease remission in patients was defined as having a Birmingham Vasculitis Activity Score of zero and not receiving glucocorticoids within 4 weeks prior to week 26. Sustained remission was defined as remission achieved at week 26 and continued at week 52, excluding any relapse of AAV.

Results at week 26 showed that compared with 70.1% of patients who received prednisone, 72.3% who received avacopan achieved remission (P <.0001 for noninferiority). At the end of week 52, 65.7% vs 54.9% of patients in the avacopan vs prednisone group achieved sustained remission, demonstrating both the noninferiority and superiority of avacopan over prednisone across the study period (P =.0066 for superiority). In addition, the avacopan group had significantly reduced glucocorticoid-related toxicity, as measured by the Glucocorticoid Toxicity Index of Cumulative Worsening Score (P =.0002).

In patients with kidney disease, the avacopan group had a mean estimated glomerular filtration rate (eGFR) increase of 7.3 mL/min/1.73 m2 at week 52, compared with an increase of 4.0 mL/min/1.73 m2 in the group treated with prednisone. Adverse events were consistent with previous AAV trials at 45.1% for the prednisone group and 42.2% for the avacopan group, with serious infections in 15.2% and 13.3% of the prednisone and avacopan groups, respectively.

These findings indicate that AAV treatment with avacopan in combination with other treatments resulted in disease remission at a rate that was noninferior to treatment with prednisone at week 26 and superior to prednisone with sustained remission at week 52. In the avacopan group, glucocorticoid-related toxicity was reduced and an increase in eGFR in patients with kidney disease was observed, suggesting that avacopan is an efficacious and relatively safe treatment for AAV.

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Merkel PA, Jayne D, Yue H, Schall T, Kelleher C, Bekker P. A randomized, double-blind, active-controlled study of avacopan in anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis. Presented at: EULAR 2020 E-Congress; June 3-6, 2020. Abstract OP0011.