Overview
Anti-neutrophil cytoplasmic (auto)antibody (ANCA)-associated vasculitis (AAV) is a rare (estimated incidence of 3 cases per 100,000 per year) form of "pauci-immune" systemic small-vessel vasculitis typified by the presence of ANCAs in the serum. The full spectrum of AAV includes granulomatosis with polyangiitis (GPA), microscopic polyangiitis (MPA), eosinophilic granulomatosis with polyangiitis (EGPA), and drug-induced AAV. AAV may be associated with necrotizing and crescentic glomerulonephritis (NCGN). Despite complex pathophysiology, studies over the past ~2 decades have identified a key role for the alternative complement pathway and, in particular, the interaction between the anaphylatoxin fragment C5a and its cognate C5aR receptor in AAV. Avacopan (formerly CCX168) is an allosteric C5aR antagonist indicated for use in AAV. Avacopan was granted FDA approval on October 8, 2021, and is currently marketed under the name TAVNEOS by ChemoCentryx, Inc. On January 19, 2022, the European Commission approved avacopan for the treatment of adult patients with severe, active granulomatosis polyangiitis (GPA) or microscopic polyangiitis (MPA) - the two main forms of ANCA-associated vasculitis - in combination with rituximab or cyclophosphamide. Avacopan was approved by Health Canada on April 20, 2022.
Indication
Avacopan is indicated for the adjunctive treatment of adult patients with severe active anti-neutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis (granulomatosis with polyangiitis and microscopic polyangiitis; GPA/MPA) in combination with standard therapy including glucocorticoids. Avacopan does not eliminate the need for glucocorticoids. In Europe, avacopan is approved for the treatment of adults with severe, active granulomatosis polyangiitis (GPA) or microscopic polyangiitis (MPA) in combination with rituximab or cyclophosphamide.
Associated Conditions
- Severe, active Granulomatosis With Polyangiitis
- Severe, active Microscopic Polyangiitis (MPA)
- Microscopic Polyangiitis (MPA)
- Severe Anti-neutrophil cytoplasmic antibody positive vasculitis
- Granulomatosis With Polyangiitis
Research Report
Avacopan (Tavneos): A Comprehensive Clinical and Pharmacological Review
1. Introduction to Avacopan
[1.1. Overview and Therapeutic Class]
Avacopan, marketed under the trade name Tavneos, is an orally bioavailable small molecule medication.[1] It functions as a selective, allosteric antagonist of the human complement 5a receptor (C5aR, also known as C5aR1 or CD88).[1] This mechanism of action positions it as a targeted immunomodulatory agent. Both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have designated Avacopan as a first-in-class medication.[1] This designation is significant as it underscores the novelty of Avacopan's therapeutic approach to ANCA-associated vasculitis (AAV). As a first-in-class agent, Avacopan targets a pathway in a manner distinct from previously available treatments, offering potential advantages for patients, particularly in addressing unmet medical needs. However, the novelty also implies that clinicians and researchers will continue to accumulate long-term real-world data to fully understand its optimal use, unique aspects of its side effect profile, and its long-term impact compared to more established therapeutic strategies.
[1.2. Approved Indications]
Clinical Trials
Title | Posted | Study ID | Phase | Status | Sponsor |
|---|---|---|---|---|---|
2025/01/03 | N/A | Recruiting | |||
2024/11/06 | Phase 2 | Not yet recruiting | |||
2024/06/21 | Phase 1 | Completed | |||
2024/03/20 | Phase 3 | Recruiting | |||
2024/01/17 | Phase 1 | Completed | |||
2023/10/10 | Phase 4 | Recruiting | |||
2023/08/22 | Phase 1 | Completed | |||
2023/08/22 | Phase 1 | Completed | |||
2023/08/14 | Phase 1 | Completed | |||
2023/08/14 | Phase 1 | Completed |
FDA Drug Approvals
Approved Product | Manufacturer | NDC Code | Route | Strength | Effective Date |
|---|---|---|---|---|---|
| ChemoCentryx, Inc. | 73556-168 | ORAL | 10 mg in 1 1 | 2/15/2022 |
EMA Drug Approvals
Approved Product | Authorization Holder | Status | Issued Date |
|---|---|---|---|
Authorised | 1/11/2022 |
HSA Drug Approvals
Approved Product | Manufacturer | Approval Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| TAVNEOS HARD CAPSULE 10MG | SIN17150P | CAPSULE, GELATIN COATED | 10mg | 12/24/2024 |
NMPA Drug Approvals
Approved Product | Company | Approval Number | Drug Type | Dosage Form | Approval Date |
|---|---|---|---|---|---|
| Avacopan Capsules | 国药准字HJ20240125 | 化学药品 | 胶囊剂 | 10/29/2024 |
PPB Drug Approvals
Approved Product | Registration No. | Company | Licence No. | Strength | Registration Date |
|---|---|---|---|---|---|
| No PPB approvals found for this drug. | |||||
TGA Drug Approvals
Approved Product | ARTG ID | Sponsor | Registration Type | Status | Registration Date |
|---|---|---|---|---|---|
| TAVNEOS avacopan 10 mg hard capsule bottle | 381053 | Medicine | A | 1/31/2023 |
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