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An Italian registry study of avacopan-treated vasculitis patients reported high remission rates, improved kidney-function measures and an average corticosteroid-dose reduction of nearly 80% by week 12. The findings add real-world evidence, but the study was small and uncontrolled and does not resolve questions about trial-data integrity or liver safety.
A multicenter Italian registry study found high rates of remission and lower corticosteroid use among patients with ANCA-associated vasculitis treated with avacopan, adding real-world evidence as the drug faces scrutiny over its pivotal trial and liver-safety concerns. The study was small and had no comparison group, so it cannot by itself establish how much benefit came from avacopan or settle the broader questions.
The researchers identified 46 patients who started avacopan in Italy between 2022 and 2024. In the group followed for at least a year, 31 of 32 patients reached clinical remission, and 55% were able to stop corticosteroids entirely, according to the report in ACR Open Rheumatology. The article describes the one-year outcomes for this subset separately from the full registry cohort.
Across the cohort, the mean daily corticosteroid dose fell from 23 milligrams at baseline to 5 milligrams by week 12, an average reduction of nearly 80%. Kidney-function measures also improved: median estimated glomerular filtration rate rose from 30.5 to 53.5 milliliters per minute per 1.73 square meters between treatment baseline and week 52. That later median remained below the 60 threshold generally used to indicate normal kidney function.
Patients also received standard immune-suppressing treatment, including rituximab, with some receiving cyclophosphamide. The study therefore reports outcomes under routine treatment conditions; it does not isolate avacopan’s effect from other therapies or compare results with a control group. The authors reported three relapses among the 46 patients, all in people with a history of relapse before starting avacopan.
Evidence Beyond the Pivotal Trial
The results matter because avacopan’s evidence base has come under scrutiny. The publication adds observations from patients treated in multiple Italian centers, rather than relying only on the registration trial whose findings were later re-adjudicated. It also reports outcomes relevant to patients and clinicians: remission, kidney function and the ability to reduce or stop long-term steroids.
Reducing steroid exposure may matter because prolonged corticosteroid use can cause serious adverse effects. But these findings do not prove that avacopan alone produced the reported changes. Without randomization or a comparison group, improvements may reflect other medicines, patient selection, disease course or a combination of factors. The registry supports further evaluation; it does not erase uncertainty about efficacy or safety.
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Why Avacopan Is Under Scrutiny
Avacopan, marketed as Tavneos, was approved in the United States in 2021 for ANCA-associated vasculitis, an autoimmune disease that inflames blood vessels and can damage organs, particularly the kidneys. The drug blocks a component of the complement pathway involved in inflammation. It is used alongside other treatments, rather than as a replacement for all immune-suppressing therapy.
Earlier in 2026, concerns emerged after data from the drug’s main registration trial were re-adjudicated after unblinding, changing an analysis that had missed its endpoint into one that met it. The New England Journal of Medicine subsequently retracted the paper reporting those findings. Separately, the FDA asked manufacturer Amgen to withdraw avacopan, citing new information about liver toxicity and the re-adjudication. Amgen had not agreed, and the FDA had not formally ordered withdrawal, according to the report. European regulators revoked the drug’s authorization in August 2026.
“Evidence from independent prospective real-world studies is particularly important to assess the effectiveness and safety of avacopan and its mechanism of action.”
— Luca Quartuccio and colleagues, as quoted in the study report
ANCA-associated vasculitis treatment
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Safety and Causation Still Unresolved
The Italian study cannot determine whether avacopan caused the reported improvements. Its small, uncontrolled design limits comparisons, and participants also received other therapies. Follow-up varied, averaging 61 weeks, with some patients followed for as long as 130 weeks.
The report provides limited information on liver outcomes, a central issue in the regulatory debate. Two patients had grade 1 liver-enzyme elevations that did not lead to treatment interruption. That observation does not settle the risk: the report cites the phase III trial as finding that 4.2% discontinued because of adverse liver effects and 5.4% had serious hepatic reactions. The registry’s size is insufficient to rule out uncommon or severe harms.
Nine of the 46 patients discontinued avacopan: three because symptoms worsened, four because of infections and two for other reasons. Seven additional patients temporarily stopped treatment. One patient died from a fungal infection after receiving rituximab for returning symptoms; the report does not establish that avacopan caused the infection or death. The study also does not resolve the dispute over the pivotal trial’s re-adjudication or the FDA’s request.
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Regulatory Review and Further Evidence
The immediate outlook depends on regulatory decisions and further evidence. In the United States, the FDA had asked Amgen to withdraw avacopan, but had not formally ordered a withdrawal as of the report’s publication; Amgen had refused the request. The report says European authorization was revoked in August 2026. The status and timing of any further U.S. action were not specified.
Clinicians and patients will need to weigh the registry findings against the trial-data concerns, known liver-safety signals and available treatment alternatives. Larger studies with longer follow-up and clear reporting of both benefits and harms would help clarify outcomes, but the Italian registry itself does not announce such a study or establish a timetable for one.
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Key Questions
What did the Italian study find about avacopan?
Among a one-year subgroup, 31 of 32 patients reached clinical remission. The study also reported lower steroid doses and improved median kidney-function measures. Because it was uncontrolled, it cannot show that avacopan alone caused those outcomes.
Did the study resolve concerns about avacopan’s pivotal trial?
No. It adds real-world observations but does not reassess the pivotal-trial data or settle concerns about its re-adjudication and the subsequent journal retraction.
What did researchers report about liver safety?
Two patients had mild, grade 1 liver-enzyme elevations that did not interrupt treatment. The registry was too small to settle liver-risk questions, and the report cites more serious hepatic findings from the phase III trial.
What is the drug’s regulatory status?
The report says the FDA had asked Amgen to withdraw avacopan but had not formally ordered withdrawal, while Amgen had refused the request. European regulators revoked its authorization in August 2026.
Source: rss
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