Real-world data show high AAV remission rates with Tavneos
Nearly all adults in 16 studies saw remission within 6 months
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Tavneos (avacopan), in combination with standard treatment, induced disease remission within six months in nearly all adults with ANCA-associated vasculitis (AAV), according to pooled results from 16 published real-world studies.
Real-world remission rates with Tavneos at six months and one year were higher than those reported in the Phase 3 ADVOCATE clinical trial (NCT02994927), data from which supported the therapy’s approvals for AAV. This difference may be attributable to more flexible dosing regimens in real-world settings, the team noted.
Still, about one-fourth of AAV patients discontinued Tavneos, primarily due to side effects, and treatment-related liver toxicity was predominantly observed in Japanese patients.
“[Tavneos] has been found to demonstrate both safety and high efficacy in the treatment of AAV in real-world settings, with remission rates exceeding and serious infection rates comparable to those observed in clinical trial data,” the researchers wrote. “However, the higher incidence of [liver toxicity] in certain populations underscores the need for careful monitoring.”
The findings were described in the review study, “Systematic Review of Efficacy and Safety of Avacopan in Real-World Clinical Practice,” published in Kidney International Reports by an international team of researchers.
Targeting the complement cascade
AAV is a group of rare autoimmune diseases characterized by self-targeting antibodies, called ANCAs, that ultimately lead to inflammation and damage to small blood vessels. The complement cascade, a component of the immune system, is known to contribute to this damaging inflammation.
Tavneos is approved for use in conjunction with standard treatments in adults with severe and active microscopic polyangiitis (MPA) and granulomatosis with polyangiitis (GPA), the two most prevalent types of AAV. Taken orally twice daily, it’s designed to suppress complement cascade activation.
In ADVOCATE, Tavneos outperformed the glucocorticoid prednisone in maintaining long-term remission and enhancing kidney function in people with AAV. It also reduced the need for glucocorticoids, which are associated with significant side effects when used for prolonged periods.
However, “real-world data on [Tavneos] remain limited, particularly in regions where AAV presents with different clinical profiles,” the researchers wrote.
The team evaluated Tavneos’ real-world efficacy and safety by systematically reviewing studies published up to September 2025. The final analysis included 16 published real-world studies, covering 447 patients with GPA or MPA treated with Tavneos. Patients ranged in age from 46 to 78, and 64% were newly diagnosed.
Most studies (75%) were retrospective in nature. Five studies were conducted in Japan, and the rest were from Europe and the U.S.
Pooled data showed that the most common therapies used to induce disease remission were high-dose glucocorticoids (61%), followed by rituximab (54%) and a combination of rituximab and cyclophosphamide (32%). Available data showed that cumulative steroid doses ranged from 700 to 3,090 mg.
The median time from diagnosis or relapse to the start of Tavneos treatment was 24 days (range, 6-54 days), and the median treatment duration ranged from 39 days (a little over a month) to 99 weeks (about two years). Complete glucocorticoid withdrawal rates varied widely across studies, from 0% to 100%, with an average of 59%.
Nine studies reported remission rates at six months, while seven reported one-year remission rates. Pooled efficacy results demonstrated that 89% of patients achieved remission after six months of Tavneos, and 87% after one year, “reflecting consistent outcomes over time in real-world settings,” the team wrote.
Few patients (7%) experienced a relapse, “without any significant difference among studies,” they added.
The pooled real-world remission rates were notably higher than those reported in ADVOCATE, with 72.3% and 65.7% of trial participants achieving remission at six and 12 months, respectively.
“This higher remission rate may be attributable to the continued use of maintenance immunosuppressive therapy following [Tavneos initiation] and to higher cumulative exposure to glucocorticoids relative to the ADVOCATE protocol,” the team wrote.
Regarding adverse events, the pooled rate of serious infections was 14%, with pneumonia (a serious lung infection) and urinary tract infections being the most frequently reported. The rate of events related to liver toxicity (hepatotoxicity) was 6% overall, with great variation between studies, but mostly observed in Japanese patients.
“It is important to perform cautious [liver] function monitoring, especially in this specific patient population,” the researchers wrote.
Additional serious adverse events potentially linked to Tavneos included low counts of immune cells or of neutrophils (a type of immune cell), gastrointestinal problems, age-related macular degeneration (an eye disease), and rash.
About one-fourth (23%) of participants discontinued Tavneos, primarily due to side effects. Other reasons included insurance-related issues, high cost, and lack of response.
“This analysis demonstrates the real-world effectiveness of [Tavneos] in AAV treatment, with serious infection rates consistent with those observed in clinical trials,” the researchers wrote. “However, the high rate of hepatotoxicity observed in the Japanese studies warrants confirmation through larger, multiethnic, controlled studies.”
