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

EMA’s human medicines committee (CHMP) has started a review of Tavneos (avacopan), following emerging information that raises questions regarding the data integrity of the Advocate study, which was the main study supporting the medicine’s marketing authorisation in the European Union. (European Medicines Agency) - "The concerns relate to how the data for the Advocate study was handled before Tavneos was authorised, which may have impacted the findings on the medicine’s effectiveness. EMA will now review all available data to assess whether this emerging information has an impact on the balance of benefits and risks of Tavneos. The Agency will then issue a recommendation on whether the marketing authorisation in the EU should be maintained, amended, suspended or revoked."

European regulatory • Eosinophilic Granulomatosis With Polyangiitis

Avacopan for severe pulmonary hemorrhage requiring extracorporeal membrane oxygenation in a patient with MPO-ANCA positive vasculitis. (PubMed, CEN Case Rep) - "A combination therapy with glucocorticoids and rituximab was initiated and avacopan was started on hospital day 8, which resulted in successful remission within six months of admission (Birmingham Vasculitis Activity Score version 3 = 0), and the beneficial effect was sustained for at least 6 months following the discontinuation of glucocorticoid withdrawal (day 156). Thus, avacopan, in combination with immunosuppressives, may not only help suppress the disease activity of AAV but also facilitate early withdrawal of glucocorticoids even in case of life-threatening respiratory failure."

Journal • ANCA Vasculitis • Hematological Disorders • Respiratory Diseases • Vasculitis • MPO

REVERSE: Avacopan Added to Standard-of-care Therapy in ANCA-associated Vasculitis With Severe Kidney Involvement (clinicaltrials.gov) - P3 | N=130 | Not yet recruiting | Sponsor: University Hospital, Toulouse

Head-to-Head • New P3 trial • ANCA Vasculitis • Colon Cancer • Vasculitis

Practical Considerations for Infection Prevention With the Clinical Use of Complement Inhibitors. (PubMed, Clin J Am Soc Nephrol) - "Currently, five complement inhibitors are approved by the United States Food and Drug Administration as effective for the treatment of kidney diseases: eculizumab, ravulizumab, avacopan, iptacopan, and pegcetacoplan. This review summarizes the infectious risks associated with complement inhibitors and highlights key clinical considerations for their safe and effective use in the treatment of kidney diseases. It is intended to serve as an accessible resource for providers utilizing these agents in clinical practice."

Journal • ANCA Vasculitis • Atypical Hemolytic Uremic Syndrome • Complement-mediated Rare Disorders • Glomerulonephritis • IgA Nephropathy • Infectious Disease • Influenza • Meningococcal Infections • Nephrology • Pneumococcal Infections • Pneumonia • Renal Disease • Respiratory Diseases • Vasculitis

Profiling of Potential Postmarket Risk Tracking and Pharmacovigilance Data for Avacopan. (PubMed, Clin Ther) - "This study reveals the potential risks of avacopan use and found significant differences in adverse events between genders and age groups. Further prospective studies are needed to validate the current findings, refine risk warnings and monitoring strategies for different populations, and explore strategies to reduce or prevent adverse reactions."

Adverse events • Journal • P4 data • Alopecia • ANCA Vasculitis • CNS Disorders • Hematological Disorders • Immunology • Infectious Disease • Insomnia • Otorhinolaryngology • Septic Shock • Sleep Disorder • Vasculitis

Targeted Delivery of Avacopan via Sortase A-Modified Extracellular Vesicles Attenuates Endotoxin-Induced Retinal Inflammation. (PubMed, Adv Healthc Mater) - "In rescue experiments, siRNA-mediated knockdown of MAPK9 reverses the downregulation of C5aR1 and pro-inflammatory cytokines, further supporting its role in mediating the therapeutic effects of MG1-EVs/Ava. The findings of this study demonstrate both the therapeutic efficacy and mechanistic basis of MG1-EVs/Ava, highlighting its potential as a promising treatment strategy for infectious uveitis."

Journal • Inflammation • Ocular Inflammation • Ophthalmology • Uveitis • C5AR1 • MAPK9

Study to Evaluate Avacopan in Combination With a Rituximab or Cyclophosphamide-containing Regimen, in Children From 6 Years to < 18 Years of Age With AAV. (clinicaltrials.gov) - P3 | N=20 | Recruiting | Sponsor: Amgen | Trial completion date: Jul 2030 ➔ Sep 2028 | Trial primary completion date: Jul 2030 ➔ Sep 2028

Trial completion date • Trial primary completion date • ANCA Vasculitis • Vasculitis

Making use of avacopan in clinical practice. (PubMed, Clin Kidney J) - "Standard remission induction therapy combines high-dose glucocorticoids (GCs) with cyclophosphamide or rituximab. Several unanswered questions remain, including the long-term safety, clinical benefit of treatment beyond 1 year and optimal GC concomitant use or even the feasibility of complete GC avoidance. Ongoing large-scale studies and international real-world evidence will be essential to define avacopan's optimal role in clinical practice, ensuring equitable access for patients with AAV."

Journal • Review • ANCA Vasculitis • Cardiovascular • Chronic Kidney Disease • Diabetes • Immunology • Infectious Disease • Metabolic Disorders • Nephrology • Osteoporosis • Renal Disease • Rheumatology • Vasculitis

Drug Repurposing Identifies Natamycin and Avacopan as Urease-Targeted Therapeutic Candidates Against Helicobacter pylori. (PubMed, Helicobacter) - "Among the top candidates, Natamycin, Zavegepant, Midostaurin, Avacopan, and Metolazone displayed significant binding affinities towards urease. Therefore, further studies are warranted to fully explore the specific mechanisms and selectivity of their individual inhibitory activities. These findings highlight Avacopan and Natamycin as promising urease-targeted drug repurposing candidates for future therapeutic development against H. pylori."

Journal • Infectious Disease

Update on antineutrophil cytoplasmic antibody vasculitis. (PubMed, Curr Opin Nephrol Hypertens) - "ANCA Vasculitis is complex with varying presentations. Through collaboration among multiple specialists, including nephrologists, rheumatologists, pulmonologists, ENT specialists, and neurologists, we are able to achieve a diagnosis and offer organ and life-saving treatments for our patients."

Journal • ANCA Vasculitis • Rare Diseases • Rheumatology • Vasculitis

Challenging manifestations of ANCA-associated vasculitis treated with avacopan: two case reports. (PubMed, Front Immunol) - "Both patients received corticosteroids, rituximab, cyclophosphamide, plasma exchange, and avacopan. Both patients achieved clinical remission with minimal glucocorticoid exposure. These cases provide insights supporting the safety and efficacy of avacopan in severe, multisystemic AAV, including rare manifestations, and highlight its potential to promote organ recovery and reduce glucocorticoid-related toxicity in high-risk presentations."

Journal • ANCA Vasculitis • Infectious Disease • Interstitial Lung Disease • Nephrology • Otorhinolaryngology • Pain • Pneumonia • Pulmonary Disease • Renal Disease • Respiratory Diseases • Vasculitis

Anti-proteinase 3 Antibody (PR3)-Anti-neutrophil Cytoplasmic Antibody (ANCA)-Positive Vasculitis: A Rare Presentation in a 90-Year-Old Female. (PubMed, Cureus) - "Treatment with corticosteroids, immunosuppressants, and avacopan resulted in improved renal function and overall resolution of symptoms, highlighting the importance of considering vasculitis as a differential diagnosis in elderly patients with multisystem involvement. This case report features the diagnostic challenges and therapeutic considerations of late-onset GPA in the geriatric population."

Journal • Acute Kidney Injury • Hematological Malignancies • Immunology • Lung Cancer • Lymphoma • Nephrology • Non-Hodgkin’s Lymphoma • Oncology • Rare Diseases • Renal Disease • Vasculitis

Complement-induced procoagulant platelets in patients with antiphospholipid antibodies (ASH 2025) - "On certain occasions sera were heat inactivated at 56º C for30 min or pre-treated with IdeS (IgG cleaving enzyme) (10000 U/ml at 37º C for 30 min), eculizumab (1mg/ml), sutimlimab (C1s inhibitor) (1.5 mg/ml) and/or danicopan (Factor D inhibitor) (100 µM). Also, onother occasions platelets were pre-incubated with antagonists of C3aR (SB290157), FcgRIIa (IV.3) or C5aR1(Avacopan) (10 µM) before serum.ResultsWe found that although activation and responsiveness to agonists of platelets from aPL+/APS patientswere like those from healthy individuals, unstimulated platelets from APS patients exhibited higherphosphatidylserine (PS) exposure (19.6±3.9% vs 4.0±1.0% controls) and P-selectin expression (15.9±2.3%vs 8.6±1.8% controls) than controls suggesting induction of procoagulant activity...Pre-incubating platelets with FcgRIIa, C3aR or C5aR1 antagonistsdid not reverse the procoagulant activity induced by APS serum.ConclusionWe demonstrate..."

Clinical • Cardiovascular • Genetic Disorders • Hematological Disorders • Immunology • Thrombosis • ANXA5 • CASP3 • CASP7 • SELP

Granulomatosis With Polyangiitis (GPA) Presenting With Painless Scleritis and Ocular Hypertension: Case Report. (PubMed, Case Reports Immunol) - "He was subsequently managed with rituximab and avacopan as per standard GPA therapy. This case highlights the importance of considering vasculitis in patients presenting with unexplained elevation of EVP, and painless scleritis, particularly when there are also extraocular complaints."

Journal • Cardiovascular • Glaucoma • Ocular Inflammation • Ophthalmology • Pain • Rare Diseases • Rheumatology • Scleritis • Vasculitis

Avacopan or Glucocorticoids for Severe Antineutrophil Cytoplasmic Autoantibody-Associated Rapidly Progressive Glomerulonephritis. (PubMed, Kidney Int Rep) - "Fifty patients received a GC regimen combined with cyclophosphamide (CYC) (30%), rituximab (RTX, 54%) or a combination of both (16%), whereas 20 patients received avacopan combined with RTX (90%) or RTX plus CYC (10%). No new safety signal was identified in the avacopan group. At months 6 and 12, patients with severe forms of ANCA-RPGN and receiving avacopan plus a short course of GCs have similar kidney recovery rates to patients receiving GC regimen."

Journal • ANCA Vasculitis • Glomerulonephritis • Inflammation • Lupus Nephritis • Nephrology • Renal Disease • Vasculitis

Mohs Surgery as a Precipitant of Bullous Disease: A Review of Reported Cases and Translational Implications (ISDS 2025) - "Five received systemic corticosteroids, often with azathioprine, doxycycline, or niacinamide; one improved with topical and intralesional therapy...Agents such as IL-1β inhibitors (e.g., anakinra), complement antagonists (e.g., avacopan), and FcRn blockers (e.g., efgartigimod) target relevant immune pathways and warrant exploration in refractory or rapidly progressive cases. While uncommon, PV, PF, and BP may emerge after MMS and mimic typical postoperative complications. Prompt biopsy and immunofluorescence facilitate early diagnosis and immunosuppressive therapy, minimizing progression and promoting wound healing."

Clinical • Review • Surgery • Bullous Pemphigoid • Contact Dermatitis • Dermatitis • Dermatology • Dermatopathology • Immunology • Infectious Disease • Pemphigus Vulgaris • IL1B

Novel Treatment Paradigms: Complement Inhibition in Antineutrophil Cytoplasmic Autoantibody Vasculitis. (PubMed, Kidney Int Rep) - "Avacopan is an oral small molecule C5a receptor antagonist approved as adjunctive therapy to standard treatments for severe active ANCA vasculitis...Although early results suggest that complement inhibitors may offer more effective and safer alternatives to established therapies, there are limitations and barriers that prevent their more widespread use. Further research is needed to better understand their efficacy and long-term safety and to inform how to optimize their integration into treatment paradigms for ANCA vasculitis."

Journal • Review • ANCA Vasculitis • Nephrology • Renal Disease • Vasculitis

Antineutrophil Cytoplasmic Antibody (ANCA) Vasculitis, Fatigue, and Treatment With Avacopan: A Case Series. (PubMed, Cureus) - "This report describes three patients who developed marked somnolence and fatigue during treatment with avacopan for antineutrophil cytoplasmic antibody (ANCA) vasculitis. Though fatigue is a common symptom in ANCA vasculitis, each of these patients reported noticeable worsening of fatigue after starting avacopan, with improvement shortly after stopping the medication. All of them had a good response to treatment of their underlying vasculitis, and none of them had other clear causes of worsening fatigue."

Journal • ANCA Vasculitis • Fatigue • Glomerulonephritis • Lupus Nephritis • Nephrology • Vasculitis

A Real-World Disproportionality Analysis of Avacopan in Anti-Neutrophil Cytoplasmic Antibodies Associated Vasculitis: Insights From FDA Adverse Event Reporting System. (PubMed, Pharmacol Res Perspect) - "This study provides the first FAERS-based pharmacovigilance assessment of Avacopan, confirming known risks and identifying novel post-approval safety signals, underscoring the need for close early-treatment monitoring and personalized strategies. Further research is warranted to confirm emerging signals and explore their mechanisms."

Adverse events • Journal • Real-world evidence • Retrospective data • Alopecia • ANCA Vasculitis • Cardiovascular • Hepatology • Immunology • Infectious Disease • Vasculitis • Venous Thromboembolism

Granulomatosis with Polyangiitis (GPA) in a Polish Tertiary Centre (2010-2025): Sex-Stratified Phenotypes, Serology, and Evolving Treatment Patterns. (PubMed, J Clin Med) - "All patients received glucocorticoids for induction; cyclophosphamide 28/50 (56.0%), rituximab 6/50 (12.0%), and mycophenolate with methotrexate 6/50 (32%). Maintenance therapy included methotrexate (78.0%), mycophenolate (64.0%), rituximab (52.0%), and azathioprine (12.0%). This Polish single-centre cohort shows an ear-nose-throat-lung-kidney (ELK)-dominant, PR3-predominant GPA phenotype and frequent but variable kidney involvement. Over 2010-2025, practice changed toward rituximab-based strategies, steroid minimisation, selective use of plasma exchange, and early avacopan uptake, with tofacitinib for maintenance therapy as a possible new therapeutic option."

Journal • ANCA Vasculitis • Chronic Kidney Disease • Inflammation • Nephrology • Ocular Inflammation • Otorhinolaryngology • Rare Diseases • Renal Disease • Respiratory Diseases • Rheumatology • Sinusitis • Vasculitis

A Rare Case of ANCA-Associated Vasculitis (AAV) with Concurrent Immune Complex (IC) Deposition (KIDNEY WEEK 2025) - "Case Description A 74-year old male with past medical history of prostate cancer (CA), non-small cell lung CA, basal cell CA, rheumatoid arthritis (on abatacept) and hypertension presented to the hospital with complaint of flank pain. He reported consuming 1.6-2g/day of ibuprofen. Home medications included nifedipine and carvedilol...He received induction treatment with rituximab, steroids and avacopan...However, the deposition of both IgG and IgA co-dominant deposits is still rare. In spite of his aggressive RPGN, we elected not to use cyclophosphamide given his recent malignancies."

Clinical • ANCA Vasculitis • Genito-urinary Cancer • Glomerulonephritis • Hypertension • IgA Nephropathy • Immunology • Inflammatory Arthritis • Lung Cancer • Lupus Nephritis • Nephrology • Non Small Cell Lung Cancer • Prostate Cancer • Renal Disease • Rheumatoid Arthritis • Rheumatology • Solid Tumor • Thrombosis • Vasculitis • MPO

ANCA Alert: A Curious Case of Amikacin-Induced Vasculitis (KIDNEY WEEK 2025) - "Introduction Drug induced-antineutrophil cytoplasmic antibody-associated vasculitis (AAV) is commonly caused by medications such as hydralazine, propylthiouracil, methimazole, and minocycline, but to date, amikacin has not been reported in the literature.We present the first case of AAV with renal involvement with amikacin as the unusual culprit...Empiric piperacillin-tazobactam and intravenous fluids were started...The patient was discharged on prednisone with plans for outpatient rituximab and avacopan...Plasma exchange and steroids resulted in significant improvement in renal function shortly after initiation which can be associated with return of renal function and creatinine apheresis. As we learn more about renal involvement in AAV, the list of causative agents expands."

Clinical • ANCA Vasculitis • Benign Prostatic Hyperplasia • Chronic Kidney Disease • Glomerulonephritis • Infectious Disease • Lupus Nephritis • Nephrology • Renal Disease • Vasculitis • MPO

Pharmacokinetics (PK) of Avacopan in Participants with ESKD Requiring Hemodialysis (HD) (KIDNEY WEEK 2025) - "Conclusion Given the PK variability of avacopan (23% to 54%), this study indicates no clinically meaningful effect of ESKD on avacopan, M1 and TAM exposures. No dose adjustment is warranted for avacopan due to ESKD or HD status."

PK/PD data • Rare Diseases • Renal Disease • Vasculitis

Patient Profiles, Treatment Patterns, and Economic Burden Among Users of Avacopan in the United States (KIDNEY WEEK 2025) - "Among those with 12-month follow-up (n=42), mean total oral prednisone-equivalent daily dose decreased from 19.8 mg to 10.6 mg between 90 days pre- and post-avacopan initiation. Conclusion A majority of users of avacopan with background immunosuppressive therapy had kidney involvement at baseline. Most patients have decreased GC exposure post-avacopan initiation."

Clinical • HEOR • Rare Diseases • Rheumatology • Vasculitis

Avacopan in Patients with ANCA-Associated Vasculitis and Severe Kidney Disease (KIDNEY WEEK 2025) - "Eighty percent received rituximab induction, and 33% received plasmapheresis. The mean time to initiation of avacopan was 43 days, with 80% successfully tapered off prednisone...Conclusion Our study demonstrated that avacopan use in AAV patients with severe kidney disease was associated with substantial eGFR recovery in non-RRT patients and had an acceptable safety profile. However, ESKD occurred in 53% of patients, and these observations warrant further study on this cohort."

Clinical • ANCA Vasculitis • Infectious Disease • Nephrology • Renal Disease • Vasculitis