Avacopan Evidence: Trial Results and Peer-Reviewed Publications

Hipa.ai Research · Source: PubMed & ClinicalTrials.gov / AACT · Last updated:

The clinical evidence base for Avacopan comprises 16 peer-reviewed publications across 10 journals, 2 pivotal-trial primary-outcome rows reported to ClinicalTrials.gov, spanning indications including Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis, and Glomerulonephritis, Membranoproliferative. Most recent publication: Efficacy and safety of avacopan for treatment of patients with ANCA-associated vasculitis receiving cyclophosphamide., RMD Open, 2025.

Top peer-reviewed publications

Curated set of pivotal-trial result papers and recent publications in high-tier journals.

  1. 2022 American College of Rheumatology/European Alliance of Associations for Rheumatology classification criteria for microscopic polyangiitis.
    Suppiah R, Robson JC, Grayson PC, et al. · Ann Rheum Dis · 2022
    PubMed: PMID 35110332 · NCT06611696 (ARRIA) · Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
  2. 2022 American College of Rheumatology/European Alliance of Associations for Rheumatology classification criteria for granulomatosis with polyangiitis.
    Robson JC, Grayson PC, Ponte C, et al. · Ann Rheum Dis · 2022
    PubMed: PMID 35110333 · NCT06611696 (ARRIA) · Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
  3. Modification and validation of the Birmingham Vasculitis Activity Score (version 3).
    Mukhtyar C, Lee R, Brown D, et al. · Ann Rheum Dis · 2009
    PubMed: PMID 19054820 · NCT06611696 (ARRIA) · Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
  4. 2012 revised International Chapel Hill Consensus Conference Nomenclature of Vasculitides.
    Jennette JC, Falk RJ, Bacon PA, et al. · Arthritis Rheum · 2013
    PubMed: PMID 23045170 · NCT06611696 (ARRIA) · Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
  5. Avacopan for the Treatment of ANCA-Associated Vasculitis.
    Jayne DRW, Merkel PA, Schall TJ, et al. · N Engl J Med · 2021
    PubMed: PMID 33596356 · NCT06611696 (ARRIA) · Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
  6. EULAR recommendations for the management of ANCA-associated vasculitis: 2022 update.
    Hellmich B, Sanchez-Alamo B, Schirmer JH, et al. · Ann Rheum Dis · 2024
    PubMed: PMID 36927642 · NCT06611696 (ARRIA) · Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
  7. Effect of Reduced-Dose vs High-Dose Glucocorticoids Added to Rituximab on Remission Induction in ANCA-Associated Vasculitis: A Randomized Clinical Trial.
    Furuta S, Nakagomi D, Kobayashi Y, et al. · JAMA · 2021
    PubMed: PMID 34061144 · NCT06611696 (ARRIA) · Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
  8. Development and initial validation of the Vasculitis Damage Index for the standardized clinical assessment of damage in the systemic vasculitides.
    Exley AR, Bacon PA, Luqmani RA, et al. · Arthritis Rheum · 1997
    PubMed: PMID 9041949 · NCT06611696 (ARRIA) · Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis

Primary-outcome results across pivotal trials

Per-arm reported values from Phase 2/3 and Phase 3 trials with results posted to ClinicalTrials.gov.

TrialIndicationPrimary endpointArmValue
NCT02994927
ADVOCATE
Anti-Neutrophil Cytoplasmic Antibody-Associated VasculitisPercentage of Subjects Achieving Disease Remission at Week 26
Week 26
Avacopan Group72.3 percentage of participants
Prednisone Group70.1 percentage of participants
NCT02994927
ADVOCATE
Anti-Neutrophil Cytoplasmic Antibody-Associated VasculitisPercentage of Subjects Achieving Sustained Disease Remission at Week 52
Week 52
Avacopan Group65.7 percentage of participants
Prednisone Group54.9 percentage of participants

Publications by year

19972025: 16 publications.

1997
1
2009
1
2013
1
2020
1
2021
2
2022
3
2024
5
2025
2

Publications by indication

Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis (14)

  • Efficacy and safety of avacopan for treatment of patients with ANCA-associated vasculitis receiving cyclophosphamide.
    RMD Open · 2025 · PMID 41052893 · NCT02994927
  • EULAR recommendations for the management of ANCA-associated vasculitis: 2022 update.
    Ann Rheum Dis · 2024 · PMID 36927642 · NCT06611696
  • Effect of Mild or Moderate Hepatic Impairment on the Pharmacokinetics of Avacopan, a Small-Molecule Complement C5a Receptor Antagonist, for the Treatment of Antineutrophil Cytoplasmic Autoantibody-Associated Vasculitis.
    Clin Pharmacol Drug Dev · 2024 · PMID 38993026 · NCT06004934
  • A phase I thorough QT/QTc study evaluating therapeutic and supratherapeutic doses of avacopan in healthy participants.
    Clin Transl Sci · 2024 · PMID 38973157 · NCT05988034
  • Efficacy and safety of avacopan in patients with ANCA-associated vasculitis receiving rituximab in a randomised trial.
    Ann Rheum Dis · 2024 · PMID 37979959 · NCT02994927

Glomerulonephritis, Membranoproliferative (1)

  • Safety and Efficacy of Avacopan in Patients with Complement 3 Glomerulopathy: Randomized, Double-Blind Clinical Trial.
    J Am Soc Nephrol · 2025 · PMID 39392695 · NCT03301467

Publications by journal

Trial-results highlights

Avacopan has been investigated in a pivotal clinical trial known as ADVOCATE, which is officially registered under the identifier NCT02994927. This study focused on patients diagnosed with Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis, a specific type of autoimmune disease. One of the primary objectives of the ADVOCATE trial was to evaluate the percentage of participants who achieved disease remission at Week 26. In the Avacopan treatment arm of NCT02994927, **72.3 percentage** of participants were reported to have achieved disease remission. In the comparator prednisone treatment arm of the same trial, NCT02994927, **70.1 percentage** of participants achieved disease remission at the Week 26 assessment point.

The ADVOCATE trial (NCT02994927) also assessed a longer-term outcome: the percentage of subjects who achieved sustained disease remission at Week 52. This endpoint aimed to understand the durability of the treatment effect. For participants receiving Avacopan in NCT02994927, **65.7 percentage** achieved sustained disease remission at Week 52. In the prednisone group of NCT02994927, **54.9 percentage** of participants achieved sustained disease remission at Week 52. These results from the ADVOCATE trial provide insights into the effects of Avacopan in managing Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis over both a short-term and a longer-term period.

All values are sourced from primary registry reporting, and individual papers should be consulted for clinical decisions.

All Avacopan publications (16)

2025 (2 papers)

  1. Efficacy and safety of avacopan for treatment of patients with ANCA-associated vasculitis receiving cyclophosphamide.
    Geetha D, Neumann T, Karras A, et al. · RMD Open · 2025 · Derived
    PubMed: PMID 41052893 · NCT02994927 (ADVOCATE) · Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
  2. Safety and Efficacy of Avacopan in Patients with Complement 3 Glomerulopathy: Randomized, Double-Blind Clinical Trial.
    Bomback AS, Herlitz LC, Kedia PP, et al. · J Am Soc Nephrol · 2025 · Derived
    PubMed: PMID 39392695 · NCT03301467 (ACCOLADE) · Glomerulonephritis, Membranoproliferative

2024 (5 papers)

  1. EULAR recommendations for the management of ANCA-associated vasculitis: 2022 update.
    Hellmich B, Sanchez-Alamo B, Schirmer JH, et al. · Ann Rheum Dis · 2024 · Trial result
    PubMed: PMID 36927642 · NCT06611696 (ARRIA) · Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
  2. Effect of Mild or Moderate Hepatic Impairment on the Pharmacokinetics of Avacopan, a Small-Molecule Complement C5a Receptor Antagonist, for the Treatment of Antineutrophil Cytoplasmic Autoantibody-Associated Vasculitis.
    Miao S, Suso P, Furst JA, et al. · Clin Pharmacol Drug Dev · 2024 · Derived
    PubMed: PMID 38993026 · NCT06004934 · Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
  3. A phase I thorough QT/QTc study evaluating therapeutic and supratherapeutic doses of avacopan in healthy participants.
    Miao S, Staehr P, Tai E, et al. · Clin Transl Sci · 2024 · Derived
    PubMed: PMID 38973157 · NCT05988034 · Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
  4. Pharmacokinetic Evaluation of the CYP3A4 and CYP2C9 Drug-Drug Interaction of Avacopan in 2 Open-Label Studies in Healthy Participants.
    Miao S, Bekker P, Armas D, et al. · Clin Pharmacol Drug Dev · 2024 · Derived
  5. Efficacy and safety of avacopan in patients with ANCA-associated vasculitis receiving rituximab in a randomised trial.
    Geetha D, Dua A, Yue H, et al. · Ann Rheum Dis · 2024 · Derived
    PubMed: PMID 37979959 · NCT02994927 (ADVOCATE) · Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis

2022 (3 papers)

  1. 2022 American College of Rheumatology/European Alliance of Associations for Rheumatology classification criteria for microscopic polyangiitis.
    Suppiah R, Robson JC, Grayson PC, et al. · Ann Rheum Dis · 2022 · Trial result
    PubMed: PMID 35110332 · NCT06611696 (ARRIA) · Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
  2. 2022 American College of Rheumatology/European Alliance of Associations for Rheumatology classification criteria for granulomatosis with polyangiitis.
    Robson JC, Grayson PC, Ponte C, et al. · Ann Rheum Dis · 2022 · Trial result
    PubMed: PMID 35110333 · NCT06611696 (ARRIA) · Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
  3. Journal Club Review of "Avacopan for the Treatment of ANCA-Associated Vasculitis".
    Soulsby WD · ACR Open Rheumatol · 2022 · Derived
    PubMed: PMID 35167187 · NCT02994927 (ADVOCATE) · Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis

2021 (2 papers)

  1. Avacopan for the Treatment of ANCA-Associated Vasculitis.
    Jayne DRW, Merkel PA, Schall TJ, et al. · N Engl J Med · 2021 · Trial result
    PubMed: PMID 33596356 · NCT06611696 (ARRIA) · Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
  2. Effect of Reduced-Dose vs High-Dose Glucocorticoids Added to Rituximab on Remission Induction in ANCA-Associated Vasculitis: A Randomized Clinical Trial.
    Furuta S, Nakagomi D, Kobayashi Y, et al. · JAMA · 2021 · Trial result
    PubMed: PMID 34061144 · NCT06611696 (ARRIA) · Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis

2020 (1 paper)

  1. Evaluation of the Safety and Efficacy of Avacopan, a C5a Receptor Inhibitor, in Patients With Antineutrophil Cytoplasmic Antibody-Associated Vasculitis Treated Concomitantly With Rituximab or Cyclophosphamide/Azathioprine: Protocol for a Randomized, Double-Blind, Active-Controlled, Phase 3 Trial.
    Merkel PA, Jayne DR, Wang C, et al. · JMIR Res Protoc · 2020 · Derived
    PubMed: PMID 32088663 · NCT02994927 (ADVOCATE) · Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis

2013 (1 paper)

  1. 2012 revised International Chapel Hill Consensus Conference Nomenclature of Vasculitides.
    Jennette JC, Falk RJ, Bacon PA, et al. · Arthritis Rheum · 2013 · Trial result
    PubMed: PMID 23045170 · NCT06611696 (ARRIA) · Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis

2009 (1 paper)

  1. Modification and validation of the Birmingham Vasculitis Activity Score (version 3).
    Mukhtyar C, Lee R, Brown D, et al. · Ann Rheum Dis · 2009 · Trial result
    PubMed: PMID 19054820 · NCT06611696 (ARRIA) · Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis

1997 (1 paper)

  1. Development and initial validation of the Vasculitis Damage Index for the standardized clinical assessment of damage in the systemic vasculitides.
    Exley AR, Bacon PA, Luqmani RA, et al. · Arthritis Rheum · 1997 · Trial result
    PubMed: PMID 9041949 · NCT06611696 (ARRIA) · Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis

Sources and methodology

This page summarizes published evidence for general reference and does not constitute medical advice. For clinical decisions, consult the linked primary publications and your healthcare provider. Data sourced from PubMed and the ClinicalTrials.gov / AACT database maintained by the Clinical Trials Transformation Initiative (CTTI).