Avacopan Has NOT Been Withdrawn from the Market
Avacopan (Tavneos) remains available and is currently undergoing regulatory review by the FDA and EMA, but has not been withdrawn. The FDA requested voluntary market withdrawal in 2024 following reports of severe drug-induced liver injury (DILI), but the manufacturer declined this request 1, 2, 3.
Current Regulatory Status
The medication's marketing authorization is under active regulatory reassessment as of April 2026, but it continues to be prescribed and is included in current clinical practice guidelines 3, 4.
- The 2024 KDIGO guidelines explicitly recommend avacopan as an alternative to glucocorticoids for remission induction in ANCA-associated vasculitis, dosed at 30 mg twice daily in combination with rituximab or cyclophosphamide 5
- The 2024 EULAR recommendations state that avacopan may be considered for induction of remission in GPA or MPA as part of a glucocorticoid-sparing strategy 6
The Hepatotoxicity Concern
The regulatory review stems from 76 worldwide DILI cases reported to the FDA (66 in Japan, 5 in the United States initially) 2. However, real-world US data from the Rheumatology Informatics System for Effectiveness registry provides important context:
- Among 238 US patients treated with avacopan, hepatic enzyme elevations were uncommon
- Only 2 confirmed DILI cases (0.8%) were identified using validated RUCAM scoring, both rated as "highly probable" causality
- One case involved histologically confirmed severe hepatocellular injury 2
The incidence rates per 100 person-years were:
- Elevated AST: 4.24 (95% CI 1.77-10.19)
- Elevated ALT: 2.67 (95% CI 0.86-8.27)
- Elevated alkaline phosphatase: 3.56 (95% CI 1.34-9.49)
- Elevated total bilirubin: 1.80 (95% CI 0.45-7.19) 2
Clinical Use Continues
Recent case reports from 2026 demonstrate ongoing clinical use with successful outcomes in severe AAV presentations, including gastrointestinal GPA with bowel perforations and MPO-AAV with diffuse alveolar hemorrhage 3, 7. These cases showed no hepatotoxicity and enabled successful glucocorticoid withdrawal.
Key Monitoring Requirements
The FDA label mandates hepatic monitoring 1:
- Measure ALT, AST, and total bilirubin before starting treatment
- Repeat testing every 4 weeks for the first 6 months
- Then every 3 months thereafter
- Interrupt treatment if ALT or AST >3× ULN
- Discontinue permanently if accompanied by bilirubin >2× ULN or clinical hepatitis
The real-world data revealed suboptimal monitoring in practice, with median time to first screening at 32 days and testing intervals averaging 125 days—far exceeding recommended frequencies 2.
Bottom Line for Practice
Avacopan remains a legitimate treatment option for AAV patients, particularly those at high risk for glucocorticoid toxicity or with lower GFR who may benefit from greater renal recovery 5. However, vigilant early hepatic monitoring is essential, and patients must be counseled about the rare but serious risk of severe DILI during the ongoing regulatory reassessment 2.