Tavneos (avacopan) / Kissei, Amgen, Otsuka, CSL Behring - LARVOL DELTA

Emergency Surgery for Gastrointestinal Complications in Patients With Granulomatosis With Polyangiitis and Microscopic Polyangiitis. (PubMed, Cureus) - "The first case involved a man in his 30s with GPA who developed massive small bowel bleeding following treatment with high-dose corticosteroids and rituximab. Emerging therapies such as avacopan may help mitigate the risk of GI complications. Early recognition and multidisciplinary management are essential to improving outcomes in patients with GI involvement in GPA/MPA."

Journal • ANCA Vasculitis • Gastroenterology • Gastrointestinal Disorder • Infectious Disease • Rare Diseases • Vasculitis

BASELINE FEATURES AND OUTCOMES AMONG PATIENTS WITH GRANULOMATOSIS WITH POLYANGIITIS OR MICROSCOPIC POLYANGIITIS ON AVACOPAN IN A COMMUNITY PRACTICE-BASED RESEARCH NETWORK IN THE U.S. (EULAR 2025) - "Disease at the time of initiation was characterized as severe active disease if patients underwent concurrent induction with rituximab or cyclophosphamide and had pre-specified life- or organ-threatening manifestations...Median (IQR) prednisone-equivalent GC dose at the time of avacopan initiation was 20.0 (10.0, 35.0) mg/day... Among patients with GPA/MPA on avacopan for at least 6 months in this real-world study, a majority (71%) on GCs at the time of avacopan initiation were able to discontinue GCs by 12 months, and the median dose of those who remained on GCs was low. Over 90% of patients had no disease worsening despite the significant reduction in GC use."

Clinical • Genetic Disorders • Immunology • Musculoskeletal Diseases • Non-melanoma Skin Cancer • Rare Diseases • Rheumatology • Skin Cancer • Solid Tumor • Vasculitis

AVACOPAN IN COMBINATION WITH RITUXIMAB ACHIEVES A HIGH REMISSION RATE IN MPA/GPA DESPITE THE LACK OF REDUCTION IN SERUM ANCA LEVELS (EULAR 2025) - "Background: The ADVOCATE trial demonstrated that avacopan, a selective oral C5a receptor antagonist, could be a potential alternative to glucocorticoid (GC) in patients with microscopic polyangiitis (MPA)/granulomatosis with polyangiitis (GPA) receiving remission induction therapy with rituximab (RTX) or cyclophosphamide [1]. In a real-world setting, avacopan reduced the GC dose and GC-related AEs, while preventing the deterioration of renal function in patients with MPA/GPA receiving rituximab for remission induction. Notably, avacopan in combination with rituximab achieved a high remission rate, despite the lack of reduction in serum ANCA levels. These findings suggest that the therapeutic effect of avacopan may be underestimated when serum ANCA is used as a biomarker."

Clinical • Combination therapy • Cardiovascular • Cataract • Diabetes • Glaucoma • Hypertension • Infectious Disease • Metabolic Disorders • Musculoskeletal Diseases • Ophthalmology • Orthopedics • Osteoporosis • Rare Diseases • Rheumatology • Vasculitis

USE OF AVACOPAN-BASED THERAPY AMONG ADULTS WITH SEVERE ACTIVE ANCA-ASSOCIATED VASCULITIS (AAV): A CROSS-SECTIONAL SURVEY OF PHYSICIANS IN GERMANY AND THE UK (EULAR 2025) - "Background: Avacopan, a selective C5aR1 inhibitor, has demonstrated efficacy and safety in its development program in patients with ANCA-associated vasculitis (AAV) (specifically granulomatosis with polyangiitis [GPA] and microscopic polyangiitis [MPA]) and has been approved in combination with rituximab or cyclophosphamide for the treatment of adults with severe active GPA/MPA. Survey results (patient record forms) suggest broad use of avacopan as part of induction therapy in adults with severe active (new-onset or relapsed) GPA/MPA in Germany and the UK. A significantly higher proportion of patients receiving avacopan- (as opposed to non-avacopan-) based therapy had depression or were overweight at their initial AAV diagnosis. Additionally, a significantly greater proportion of these patients had multiple organ involvement, lung or skin involvement, presented with ‘general’ or skin symptoms, and/or had higher CRP values at their most recent induction."

Clinical • ANCA Vasculitis • Cardiovascular • CNS Disorders • Depression • Diabetes • Dyslipidemia • Hypertension • Metabolic Disorders • Mood Disorders • Nephrology • Obesity • Psychiatry • Rare Diseases • Rheumatology • Type 2 Diabetes Mellitus • Vasculitis

SAFETY PROFILE OF AVACOPAN IN ANCA-ASSOCIATED VASCULITIS: INSIGHTS FROM THE JAPANESE MULTICENTER J-CANVAS STUDY (EULAR 2025) - "The imbalance of clinical characteristics between the two groups was adjusted by overlap weighting with propensity scores calculated from age, sex, diagnosis, diabetes mellitus, smoking, pulmonary involvement, renal involvement, BVAS, C-reactive protein, intravenous cyclophosphamide use, rituximab use, methylprednisolone pulse use, the initial dose of prednisolone, and use of trimethoprim-sulfamethoxazole. This study provides real-world data on the safety profile of avacopan in Japanese patients with AAV. The risk of serious infections in the avacopan-treated patients was comparable to non-avacopan-treated patients. However, liver dysfunction emerged as the most frequent SAE of avacopan."

Clinical • ANCA Vasculitis • Diabetes • Hepatology • Infectious Disease • Inflammation • Liver Failure • Metabolic Disorders • Rare Diseases • Vasculitis • CRP

EFFICACY OF AVACOPAN AS INDUCTION THERAPY IN ANCA-ASSOCIATED VASCULITIS: A JAPANESE MULTICENTER COHORT STUDY (J-CANVAS STUDY) (EULAR 2025) - " This study utilized data from the Japanese AAV patient registry (J-CANVAS), specifically focusing on cases of newly diagnosed or severely relapsed microscopic polyangiitis (MPA) or granulomatosis with polyangiitis (GPA) registered between January 2017 and March 2023, who received intravenous cyclophosphamide (IVCY) or rituximab (RTX) as induction therapy...Azathioprine was the most commonly used concomitant medication in both groups during remission maintenance, at 26.4% in the Avacopan group and 44.2% in the control group (p=0.16)... In Japan, Avacopan is primarily utilized for AAV patients with severe renal dysfunction, demonstrating comparable remission rates to conventional therapy. On the other hand, renal dysfunction remained over the 48-week observation period after remission induction, regardless of whether Avacopan was used or not. Notably, it enabled a significant and earlier reduction in PSL use, potentially mitigating long-term glucocorticoid-related adverse..."

Clinical • ANCA Vasculitis • Infectious Disease • Inflammation • Nephrology • Pain • Rare Diseases • Renal Disease • Vasculitis

Targeted B-cell therapy by anti-CD19 CAR T cells induces stable disease remission in treatment-refractory ANCA-associated vasculitis (EULAR 2025) - "Case presentation: A 52-year-old male with 20 years of severe, treatment-refractory (cyclophosphamide, rituximab, mycophenolate mofetil, azathioprine and methotrexate) PR3-ANCA+ AAV involving multiple organs (lungs, kidneys, joints, skin, sinonasal tract, eyes) presented with fever, weight loss, myalgia, arthralgia, exertional dyspnea, and productive cough (BVAS 6) despite repetitive treatment with rituximab over the past 10 years...Reinduction therapy with rituximab and avacopan partially improved dyspnea and stabilized weight, but myalgia and arthralgia persisted (BVAS 2). Due to relapsing disease, he received 1x10 8 anti-CD19 CAR T cells following lymphodepletion with cyclophosphamide and fludarabine...He developed grade 1 CRS managed with 3 doses of tocilizumab and grade 3 neutropenia, resolved with filgrastim...At 7 months, the patient remained symptom-free (BVAS 0) without immunosuppressive treatment despite the return of CD19+ B cells. Learning points for clinical..."

CAR T-Cell Therapy • Clinical • ANCA Vasculitis • Cough • Hematological Disorders • Infectious Disease • Musculoskeletal Pain • Neutropenia • Pain • Pulmonary Disease • Respiratory Diseases • Vasculitis

Glucocorticoid free remission in maintenance treatment of anca associated vasculitis (AAV) after discontinuation of avacopan - a unicentre non-interventional prospective observational study of 34 patients over 18 months (EULAR 2025) - " From March 2022 to January 2024, 86 patients with AAV were treated at Minden University Hospital (in the Department of Rheumatology and the Department of Nephrology), 34 of whom underwent remission induction with cyclophosphamide (CYC), rituximab (RTX) or a combination of CYC/RTX and avacopan. Following the introduction of avacopan into the AAV treatment regime, a significant number of patients achieved GC free remission within 18 months, even after stopping avacopan. However, a longer observation period and larger cohorts should confirm this observation."

Clinical • Observational data • ANCA Vasculitis • Infectious Disease • Nephrology • Osteoporosis • Rheumatology • Vasculitis

Recent glucocorticoid-sparing strategies lead to better prognosis for ANCA-associated vasculitis: Insights from the multicenter REVEAL cohort study (EULAR 2025) - "Background: Anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) continues to carry a poor prognosis, and its treatment has historically relied on glucocorticoids (GC) and cyclophosphamide, forcing patients to endure burdensome side effects, particularly infections. Recently, novel molecularly targeted therapies, including mepolizumab and avacopan, have been introduced... Although MPA remains associated with the highest mortality among AAV subtypes, the recent widespread adoption of GC-sparing strategies appears to have improved the overall prognosis of AAV."

Clinical • ANCA Vasculitis • Eosinophilic Granulomatosis With Polyangiitis • Infectious Disease • Langerhans Cell Histiocytosis • Rare Diseases • Vasculitis • CRP • MPO

Efficacy and Safety of Avacopan for Treatment of Patients with ANCA-Associated Vasculitis Receiving Cyclophosphamide (EULAR 2025) - " Patients in the ADVOCATE trial received, at investigators’ discretion, either CYC followed by azathioprine (or mycophenolate mofetil if azathioprine was not tolerated) or rituximab. The analysis of the subgroup of patients who received CYC followed by azathioprine (or mycophenolate mofetil) in the ADVOCATE trial suggests similar rates of remission in the avacopan group compared with the prednisone taper group. Use of avacopan with a markedly reduced GC regimen was associated with a numerically lower relapse rate, greater improvement in eGFR, faster reduction in UACR, lower GC-related toxicity, and greater improvements in HRQoL vs a prednisone taper. Safety appeared similar between the two treatment groups."

Clinical • ANCA Vasculitis • Infectious Disease • Pneumonia • Rare Diseases • Respiratory Diseases • Vasculitis • MPO

Interim Report on Post-Marketing Surveillance of avacopan, a C5a Receptor antagonist, for Microscopic Polyangiitis and granulomatosis with polyangiitis-1st Report- (EULAR 2025) - "The PMS study revealed the safety and effectiveness of avacopan for 6 months after commencing treatment in patients with MPA and GPA in the real-world. Patient characteristics at baseline Adverse events and adverse drug reactions"

P4 data • Gastrointestinal Disorder • Hepatology • Infectious Disease • Rare Diseases • Vasculitis

Complement fraction C5a, C5a receptor 1, and its associated signaling signature are overexpressed in giant-cell arteritis lesions (EULAR 2025) - "4) To interrogate the transcriptomic results searching for transcripts regulated by C5a receptor signaling and the impact of blocking IL-6 receptor with tocilizumab (TCZ), the only targeted therapy currently approved for GCA, on their expression...Exploring the transcriptomic and functional impact of avacopan on cultured temporal arteries is in progress. Complement pathways are up regulated in GCA tissue and C5a is present in the supernatant of cultured arteries indicating local activation of the alternative pathway of the complement system. C5aR1 is expressed in GCA-involved arteries by inflammatory cells but also by endothelial cells and stromal cells, suggesting a function in vascular remodeling. Transcripts regulated by C5aR1 (but not regulated by other cytokines present in GCA) are overexpressed in GCA and are not modulated by TCZ."

ANCA Vasculitis • Giant Cell Arteritis • Immunology • Inflammation • C5AR1 • CSF2 • IFNG • IL6 • PTGER3 • SPI1 • STAT1 • STAT3 • TLR2

Early Access Program (EAP) of Avacopan for the Treatment of ANCA-Associated Vasculitis (AAV): The Italian Multicenter Prospective Real-World Experience Beyond 52 Weeks (EULAR 2025) - "This study highlights the early and sustained efficacy, safety, and improvements in quality-of-life scores associated with avacopan treatment in the real-world management of AAV in a cohort of Italian patients. Avacopan enables early clinical remission, CS reduction, and improvements in renal function parameters. Treatment discontinuation occurred in 20% of patients due to relapses, infections, or comorbidities; however, the continuation of treatment beyond one year in nearly half of patients suggests that avacopan is well-tolerated for long-term disease control."

Clinical • Real-world • Real-world evidence • ANCA Vasculitis • Glomerulonephritis • Hematological Disorders • Infectious Disease • Inflammation • Lupus Nephritis • Nephrology • Renal Disease • Rheumatology • Vasculitis

Comparing avacopan use in patients with GPA during induction phase with Rituximab and prednisone: A retrospective study using the TriNetx database (EULAR 2025) - "In patients who present with organ or life-threatening disease, it is recommended to start induction therapy with steroids and either Cyclophosphamide or Rituximab. Despite being a new medication with reported side effects related to the liver, avacopan seems to be well tolerated, however some of the infections explored in this study seem to be more prevalent in the group receiving avacopan, however, it is unclear if this is the result of the medication itself or a confounding factor as the cohort is smaller than the group that was receiving just Rituximab and prednisone. Furthermore, it does not appear that the implementation of Avacopan decreases the toxicity resulting from steroids as neither of the groups reported a significant statistical difference when it came to some of the explored side effects in these. In addition, it does not appear that these medication impacts cardiovascular risk negatively as there was no increase on MACE risk for any of these patients,..."

Retrospective data • Cardiovascular • Diabetes • Endocrine Disorders • Glomerulonephritis • Hepatitis B • Hepatitis C • Hepatology • Herpes Simplex • Herpes Zoster • Infectious Disease • Inflammation • Lupus Nephritis • Metabolic Disorders • Nephrology • Rare Diseases • Renal Disease • Respiratory Diseases • Tuberculosis • Varicella Zoster • Vasculitis

Serum C3 levels at diagnosis in renal antineutrophil cytoplasmic antibody-associated vasculitis: predictors of lung disease and poorer renal survival (EULAR 2025) - "This was further corroborated with the efficacy of Avacopan, a selective C5a receptor inhibitor used as a corticosteroids reduction strategy in the treatment of AAV [1]... Complement activation in AAV has been associated to renal outcomes, but our results also highlight a possible role of complement in the pathophysiology of lung disease, with low sC3 levels as an independent predictor of lung involvement and of the occurrence of alveolar hemorrhage. Patients with low sC3 levels without real hypocomplementemia had a higher risk of requiring acute dialysis and a poorer renal prognosis, expanding the existent evidence on the relevance of the alternative complement pathway activation in AAV patients."

ANCA Vasculitis • Hematological Disorders • Pulmonary Disease • Respiratory Diseases • Vasculitis

Three years of avacopan treatment in patients with ANCA-associated vasculitis (EULAR 2025) - "Of the 6 patients with <1 year avacopan treatment, 5/6 patients received a steroid-taper and 4/6 (67%) received rituximab maintenance in the first year of follow-up. The median prednisone dosage was 23 [16-29] mg/day at baseline, 3.8 [0.6-6.9] mg/day after 6 months and 2.5 [0-5] mg/day after 12 months...In patients were avacopan was discontinued 3/6 patients continued steroids, 3/6 received RTX maintenance treatment and 1/6 received azathioprine treatment... Our study is the first descriptive analysis of three years avacopan treatment in severe AAV patients in which we observed lower hospitalization and infection rates when compared to stopping avacopan treatment wihtin 1 year. These data warrant further confirmation in larger, observational studies including the currently recruiting AVAC-EUR study."

Clinical • ANCA Vasculitis • Infectious Disease • Vasculitis

Pediatric ANCA-Associated Vasculitis Study of Avacopan Efficacy, Pharmacokinetics, and Safety: A Phase 3 Trial in Progress (EULAR 2025) - P3 | "Objectives: Here we describe a phase 3, open-label, single-arm trial in progress designed to evaluate the efficacy, pharmacokinetics, and safety of avacopan when combined with a rituximab (RTX)- or cyclophosphamide (CYC)-containing regimen in pediatric patients with newly diagnosed or relapsing active GPA/MPA (NCT06321601)... This trial will evaluate the primary endpoints of remission at week 26 (either a PVAS of 0 with a prednisone-equivalent dose of ≤ 0.2 mg/kg/day [maximum dosage of 10 mg/day] within the past 4 weeks or a PVAS of 0 for at least 4 weeks, provided the last PVAS assessment is within the prior 8 weeks, irrespective of the GC dose being received) and sustained remission at week 52 (defined as PVAS-based remission at week 26 and week 52 without relapses [worsening of disease after previously achieving remission involving 1 or 2 PVAS nonmajor items present for 2 consecutive study visits, ≥ 1 PVAS major item, or ≥ 3 PVAS nonmajor items] between the two..."

Clinical • P3 data • PK/PD data • ANCA Vasculitis • Inflammation • Pediatrics • Rare Diseases • Renal Disease • Vasculitis

AvacoStar, a real-world evidence study of avacopan in ANCA-associated vasculitis – baseline characteristics of initial patients (EULAR 2025) - P | "In the pivotal phase 3 ADVOCATE study, avacopan was non-inferior to prednisone-taper for achieving remission at Week 26, superior in sustaining remission at Week 52, and was associated with improved estimated glomerular filtration rate from baseline. At the time of data collection, the majority of baseline characteristics are comparable in general between the cohorts with some imbalances in duration since AAV diagnosis and organ involvement. There is a higher proportion of patients that received rituximab plus cyclophosphamide for induction in the avacopan cohort. The initial findings indicate that the outcomes of the study could provide valuable and applicable information about the safety and usage patterns of avacopan."

Clinical • HEOR • Real-world • Real-world evidence • ANCA Vasculitis • Hepatology • Infectious Disease • Liver Failure • Oncology • Rare Diseases • Vasculitis

Avacopan may not independently reduce ANCA titres in ANCA-Associated vasculitis: a retrospective analysis of 57 patients. (PubMed, Rheumatology (Oxford)) - No abstract available

Journal • Retrospective data • ANCA Vasculitis • Vasculitis

Abatacept and multiple therapeutic bronchoscopies as salvage therapy for refractory tracheobronchial inflammation and stenosis in GPA. (PubMed, BMJ Case Rep) - "Rituximab (RTX), in combination with glucocorticosteroids, is a well-established and effective alternative to cyclophosphamide (CYC) for inducing remission in GPA...She was subsequently induced successfully with CYC but attempts to transition to another maintenance immunosuppressive agent failed, despite the addition of avacopan...We review the literature on treatment approaches to GPA TBD. In patients with severe, refractory GPA TBD, abatacept should be considered as a therapeutic option."

Journal • Inflammation • Rare Diseases • Vasculitis

3-months short course of avacopan in ANCA vasculitis with severe clinical involvement. A real-life experience. (ERA 2025) - "Background and Aims:Over the last decade, seminal studies allowed the implementation of rituximab as induction therapy (instead of or in addition to cyclophosphamide) and the decrease of cumulative steroid doses in the treatment of ANCA- associated vasculitis (AAV)...The ADVOCATE study's maintenance regimen was criticised because azathioprine was used rather than rituximab...All patients received rituximab alone as induction therapy (1 g x 2 n= 4/12, 375m2 x 4, n= 8/12) on top of glucocorticoid boluses (mean dose 1416 mg), followed by oral prednisone according to the PEVIVAS low-dose regimen (Figure 1a), with a mean dose of 5 mg or less achieved at 126 days... In a series of severe AAV with renal involvement, the combination of rituximab with a 3-month short course of avacopan and a PEXIVAS low-dose glucocorticoid regimen was safe and allowed remission without progressive disease in patients with severe renal involvement and no relapse was observed when rituximab was..."

Clinical • ANCA Vasculitis • Cardiovascular • Renal Disease • Vasculitis

A cluster analysis of a monocentric cohort of ANCA Vasculitis: implications on induction – maintenance therapeutic strategies and role of repeat renal biopsy (ERA 2025) - "Over the years quite a consensus has been achieved on induction therapy, mainly based on the association of glucocorticoids with rituximab (RTX) or cyclophosphamide (CYC) or their combination. Avacopan has been recently approved as a steroid sparing strategy, while the role of plasmapheresis is more and more debated... Treatment of AAVs has improved over the last decade. As AAVs are typically characterized by a relapsing course, and to optimize tailored approaches physicians should be aware of unique features that may influence clinical response. Data from the present cohort of patients followed-up over the last 10 years confirmed the effectiveness of a personalized treatment and allowed the elaboration of an algorithm showing how, once a clinical remission is achieved, the AAV patient is managed according to our school strategy."

Biopsy • Clinical • ANCA Vasculitis • Inflammation • Renal Disease • Vasculitis

Use of avacopan-based therapy among adults with severe active ANCA-associated vasculitis (AAV): a cross-sectional survey of physicians in Germany and the UK (ERA 2025) - "Background and Aims:Avacopan, a selective C5aR1 inhibitor, has demonstrated efficacy and safety in its development program in patients with ANCA-associated vasculitis (AAV) (specifically granulomatosis with polyangiitis [GPA] and microscopic polyangiitis [MPA]) and has been approved in combination with rituximab or cyclophosphamide for the treatment of adults with severe active GPA/MPA. Survey results (patient record forms) suggest broad use of avacopan as part of induction therapy in adults with severe active (new-onset or relapsed) GPA/MPA in Germany and the UK. A significantly higher proportion of patients receiving avacopan- (as opposed to non-avacopan-) based therapy had depression or were overweight at their initial AAV diagnosis. Additionally, a significantly greater proportion of these patients had multiple organ involvement, lung or skin involvement, presented with 'general' or skin symptoms, and/or had higher CRP values at their most recent induction."

Clinical • ANCA Vasculitis • Cardiovascular • CNS Disorders • Depression • Diabetes • Dyslipidemia • Hypertension • Metabolic Disorders • Mood Disorders • Nephrology • Obesity • Psychiatry • Rare Diseases • Rheumatology • Type 2 Diabetes Mellitus • Vasculitis

Three years of avacopan treatment in patients with ANCA-associated vasculitis (ERA 2025) - "Of the 6 patients with <1 year avacopan treatment, 5/6 patients received a steroid-taper and 4/6 (67%) received rituximab maintenance in the first year of follow-up. The median prednisone dosage was 23 [16-29] mg/day at baseline, 3.8 [0.6- 6.9] mg/day after 6 months and 2.5 [0-5] mg/day after 12 months...In patients were avacopan was discontinued 3/6 patients continued steroids, 3/6 received RTX maintenance treatment and 1/6 received azathioprine treatment... Our study is the first descriptive analysis of three years avacopan treatment in severe AAV patients in which we observed lower hospitalization and infection rates when compared to stopping avacopan treatment <1 year. These data warrant further confirmation in larger, observational studies including the currently recruiting AVAC-EUR study."

Clinical • ANCA Vasculitis • Infectious Disease • Vasculitis

An 18 month outcome survey of the utilisation, accessibility and value of the Eastern Network for Kidney Inflammatory Disease (ENKID) virtual multidisciplinary team meeting (MDT). (UKKW 2025) - "34 responders reported receiving HCD approval for avacopan (74%), rituximab (56%), targeted-release budesonide (38%), belimumab (29%) and voclosporin (26%) (Figure 2). Overall 15 centres with 75 health care professional attendees suggests high accessibility; however, ongoing work will describe geographical access to ENKID and HCD use across the East of England, to ensure that discrepancies in access are identified and resolved. With further HCDs on the horizon for autoimmune kidney diseases, robust services and networks such as ENKID are essential to ensure specialist advice and equity of access to HCDs."

ANCA Vasculitis • Glomerulonephritis • Immunology • Inflammation • Inflammatory Arthritis • Nephrology • Renal Disease • Rheumatology • Vasculitis