Fabhalta (iptacopan) / Novartis - LARVOL DELTA

EFFICACY AND SAFETY OF IPTACOPAN IN PAROXYSMAL NOCTURNAL HEMOGLOBINURIA: A REAL-WORLD STUDY OF 37 PATIENTS IN CHINA (EBMT 2026) - " Among the 37 patients, 3 had received prior eculizumab and the remainder were C5 inhibitor-naive. Iptacopan treatment achieved substantial clinical improvement in both classical PNH and PNH/BMF patients, as evidenced by transfusion-free hemoglobin rises and normalization of hemolytic markers in the majority."

Clinical • Real-world • Real-world evidence • Aplastic Anemia • Complement-mediated Rare Disorders • Hematological Disorders • Musculoskeletal Diseases • Musculoskeletal Pain • Orthopedics • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

ORAL IPTACOPAN DEMONSTRATES EFFICACY AS SALVAGE THERAPY FOR HIGH-RISK TRANSPLANT-ASSOCIATED THROMBOTIC MICROANGIOPATHY: FIRST REAL-WORLD EXPERIENCE (EBMT 2026) - "Prior salvage therapies were extensive, including calcineurin inhibitor withdrawal (n=19), rituximab (n=11), and eculizumab (n=9), plasma exchange (n=7), and defibrotide (n=4). In conclusion, the oral complement inhibitor iptacopan demonstrated significant clinical efficacy as salvage therapy in high-risk TA-TMA patients, irrespective of prior eculizumab exposure."

Clinical • Real-world • Real-world evidence • Acute Graft versus Host Disease • Bone Marrow Transplantation • Cerebral Hemorrhage • Graft versus Host Disease • Hematological Disorders • Immunology • Infectious Disease • Meningococcal Infections • Pneumonia • Respiratory Diseases • Transplantation • Transplantation Associated Thrombotic Microangiopathy • CFB

EFFICACY AND SAFETY OF IPTACOPAN IN PAROXYSMAL NOCTURNAL HEMOGLOBINURIA: A REAL-WORLD STUDY OF 37 PATIENTS IN CHINA (EBMT 2026) - " Among the 37 patients, 3 had received prior eculizumab and the remainder were C5 inhibitor-naive. Iptacopan treatment achieved substantial clinical improvement in both classical PNH and PNH/BMF patients, as evidenced by transfusion-free hemoglobin rises and normalization of hemolytic markers in the majority."

Clinical • Real-world • Real-world evidence • Aplastic Anemia • Complement-mediated Rare Disorders • Hematological Disorders • Musculoskeletal Diseases • Musculoskeletal Pain • Orthopedics • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

The complement factor B inhibitor iptacopan for relapsed and refractory immune thrombotic thrombocytopenic purpura. (PubMed, Thromb J) - No abstract available

Journal • Hematological Disorders • Thrombocytopenic Purpura • CFB

ORAL IPTACOPAN THERAPY IN C3 GLOMERULOPATHY SHOWS CONSISTENT STABILIZATION OF EGFR SLOPE ACROSS PATIENT SUBGROUPS (ISN-WCN 2026) - P3 | "DK was scientific founder of Gyroscope Therapeutics; Received consultancy income from Achillion, Alexion, AstraZeneca,Catalyst, Chemocentryx, Novartis, Roche, and Silence Therapeutics; Received grants or contracts from Gyroscope Therapeutics, Kidney Research UK, Macular Society, Medical Research Council, and Wellcome Trust; His spouse works for GSK. ASB.I did not use generative AI and AI-assisted technologies in the writing process."

Clinical • Complement-mediated Rare Disorders • Glomerulonephritis • Lupus Nephritis • EGFR

SUCCESSFUL TRANSITION FROM ECULIZUMAB TO IPTACOPAN IN A PEDIATRIC CASE OF C3 GLOMERULOPATHY REFRACTORY TO IMMUNOSUPPRESSIVE THERAPY (ISN-WCN 2026) - "Treatment with corticosteroids, RAAS inhibitors, and mycophenolate mofetil was ineffective. No serious infections or adverse events occurred.Conclusion In this case, downstream complement blockade with eculizumab provided limited efficacy in C3G with upstream alternative pathway dysregulation. In contrast, upstream factor B inhibition with iptacopan improved renal parameters, suggesting that targeting the alternative pathway upstream may represent a more pathophysiology-based treatment strategy for similar patients."

Clinical • Complement-mediated Rare Disorders • Glomerulonephritis • Infectious Disease • Lupus Nephritis • Pediatrics

SUCCESSFUL TREATMENT OF A PATIENT WITH C3 GLOMERULOPATHY (C3G) WITH IPTACOPAN: 10 MONTHS EXPERIENCE (ISN-WCN 2026) - "From this point-of view, the current case report , shows that iptacopan is highly effective regarding the reduction of proteinuria and recovery and stabilization of kidney function and is well tolerated. Our case report suggests that even under the 10-month monotherapy, iptacopan may become a promising oral treatment option for C3G."

Clinical • Complement-mediated Rare Disorders • Glomerulonephritis • Hematological Disorders • Infectious Disease • Influenza • Lupus Nephritis • Meningococcal Infections • Pneumococcal Infections • Respiratory Diseases

EARLY INSIGHTS INTO CHARACTERISTICS AND TREATMENT PATTERNS OF US PATIENTS WITH COMPLEMENT 3 GLOMERULOPATHY WHO WERE PRESCRIBED IPTACOPAN: THE APPRISE-C3G DATA PLATFORM (ISN-WCN 2026) - "This interim analysis provides early insights into demographic and clinical characteristics and treatment patterns of patients enrolled in APPRISE-C3G to date.Methods The APPRISE data platform captures retrospective, longitudinal, deidentified patient-level data from patients with C3G, immunoglobulin A nephropathy (IgAN), or paroxysmal nocturnal hemoglobinuria treated with iptacopan and/or atrasentan. Median (IQR) latest reported pre-index urine protein-to-creatinine ratio (n=7) was 4.2 (2.7–7.3) g/g.Conclusion This interim analysis of APPRISE-C3G provides first insights into characteristics and treatment patterns of US patients with C3G prescribed iptacopan in a real-world setting. Recruitment for APPRISE-C3G is ongoing and will provide further valuable information about this patient population over time.I have potential conflict of interest to disclose.This study was funded by Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA.I did not use generative AI and..."

Clinical • Complement-mediated Rare Disorders • Glomerulonephritis • Hematological Disorders • IgA Nephropathy • Lupus Nephritis • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

C3 GLOMERULOPATHY WITH AUTOIMMUNE-LIKE FEATURES: A 12-YEAR COURSE AND RATIONALE FOR IPTACOPAN THERAPY (ISN-WCN 2026) - "Methotrexate was changed to mycophenolate mofetil (MMF) in year 4 (age 17) for relapsing arthralgia and proteinuria...Hydroxychloroquine (HCQ) was administered at year 7 (age 20) for skin and joint manifestations...During years 10-11 (ages 23-24), baricitinib was introduced after belimumab withdrawal but was discontinued at year 12 (age 25) owing to limited efficacy and infection risk...Despite multi-agent immunosuppression, the patient remained steroid-dependent, indicating complement-driven disease activity. Iptacopan, an oral factor B inhibitor, offers a rational steroid-sparing option for complement-driven C3G activation unresponsive to conventional therapy."

Complement-mediated Rare Disorders • Glomerulonephritis • Herpes Zoster • Immunology • Infectious Disease • Inflammatory Arthritis • Lupus • Musculoskeletal Pain • Systemic Lupus Erythematosus • Varicella Zoster • CTLA4

REBIOPSY-GUIDED LONG-TERM MANAGEMENT AND IPTACOPAN INTRODUCTION IN A STEROID-DEPENDENT CASE OF C3 GLOMERULOPATHY WITH CYCLOSPORINE NEPHROTOXICITY (ISN-WCN 2026) - "Subsequently, mycophenolate mofetil (MMF 1–1.5 g/day) was added. Iptacopan, an oral factor B inhibitor, reduced proteinuria by ∼40% and normalized complement activity within 12 months in the phase 3 APPEAR-C3G trial, supporting both its mechanistic rationale and clinical applicability in this case. The transition marks a strategic shift toward targeted factor B inhibition aiming for sustained disease control with minimized exposure to conventional immunosuppression."

Clinical • Complement-mediated Rare Disorders • Fibrosis • Glomerulonephritis • Immunology • Nephrology

UPDATE TO THE LONG-TERM EFFICACY AND SAFETY OF IPTACOPAN IN C3 GLOMERULOPATHY: 39-MONTH PHASE 2 EXTENSION STUDY DATA (ISN-WCN 2026) - P3 | "This study is sponsored by Novartis Pharma AG. Professional medical writing assistance was provided by Carol Crawford (Novartis Ireland Ltd., Dublin, Ireland) and Andrew Jobson (Novartis Pharmaceuticals UK Ltd., London UK), funded by Novartis Pharma AG.I did not use generative AI and AI-assisted technologies in the writing process."

Clinical • P2 data • Complement-mediated Rare Disorders • Glomerulonephritis

CLINICAL EFFICACY OF PEGCETACOPLAN VERSUS IPTACOPAN IN PATIENTS WITH C3 GLOMERULOPATHY: INDIRECT TREATMENT COMPARISONS (ISN-WCN 2026) - P3 | "The findings from this analysis may help guide clinicians managing patients with this rare condition. This abstract was also submitted for the ASN Kidney Week 2025 congress.I have potential conflict of interest to disclose.Bradley P Dixon reports consultancy from Apellis, Novartis, Alexion, AstraZeneca, Arrowhead, Calliditas; Andrew S Bomback reports consultancy and/or honoraria from Achillion, Alexion, Amgen, Apellis, Aurinia, Calliditas, Catalyst, Genentech, GSK, Kezar, Novartis, Otsuka, Q32, Silence Therapeutics, UpToDate; Carly Rich is an employee of Sobi; Mingyi Huang is an employee of Apellis; Piotr Wojciechowski is an employee of Clever Access; Rose Chang is an employee of the Analysis Group, Inc.; Fernando Caravaca-Fontán reports consultancy and/or honoraria from Alexion, Apellis, AstraZeneca, Novartis, Otsuka, Roche, Sobi, Vifor; Fadi Fakhouri reports consultancy and/or honoraria from Alexion, Apellis, AstraZeneca, Novartis, Roche, Sanofi, Sobi.I did..."

Clinical • Complement-mediated Rare Disorders • Glomerulonephritis

EFFICACY AND SAFETY OF IPTACOPAN IN PARTICIPANTS WITH C3 GLOMERULOPATHY: C3G EXTENSION TRIAL INTERIM RESULTS FROM THE PHASE 3 APPEAR-C3G PARTICIPANTS (ISN-WCN 2026) - P3 | "Declaration of funding and interests: This study is funded by Novartis Pharma AG. Professional medical writing assistance was provided by Mayuri Shinde (Novartis, Hyderabad, India) and Andrew Jobson (Novartis, Dublin, Ireland) and funded by Novartis Pharma AG.I did not use generative AI and AI-assisted technologies in the writing process."

Clinical • P3 data • P3 data: top line • Complement-mediated Rare Disorders • Glomerulonephritis

SUCCESSFUL TREATMENT OF RECURRENT DENSE DEPOSIT DISEASE POST KIDNEY TRANSPLANT WITH IPTACOPAN FOLLOWING ECULIZUMAB THERAPY (ISN-WCN 2026) - "To our knowledge, no prior case reports have mentioned successful use of Iptacopan therapy after eculizumab therapy in post-transplant C3GN or DDD.Conclusion Iptacopan could potentially be a well tolerated option in transplant patients with DDD or C3GN, in cases with intolerance or failure to eculizumab. Further studies are needed to define its exact role."

Chronic Kidney Disease • Complement-mediated Rare Disorders • Nephrology • Renal Disease • Transplantation

IPTACOPAN TREATMENT IN PATIENTS WITH C3 GLOMERULOPATHY (C3G): 12-MONTH RESULTS FROM THE MANAGED ACCESS PROGRAM (ISN-WCN 2026) - "Professional medical writing assistance was provided by Abhinaya Nayak (Novartis Healthcare Pvt Ltd), funded by Novartis Pharma AG. CN is associate Director for Molecular Otolaryngology and Renal Research Laboratory; receives NIH grant support (2R01DK110023-07); serves on advisory boards for Novartis, Apellis, Vertex and Alexion; participates as a site investigator for Novartis and Apellis; is a member of the data safety monitoring board for Purespring; serves as Chair of a data safety monitoring board for FIT4KID; and receives author royalties from UpToDateI did not use generative AI and AI-assisted technologies in the writing process."

Clinical • Complement-mediated Rare Disorders • Glomerulonephritis • Otorhinolaryngology

Efficacy and Safety of Iptacopan in Patients With IgA Nephropathy (IgAN): Final 24-month Results From the Phase III APPLAUSE-IgAN Study (ISN-WCN 2026) - No abstract available

Clinical • Late-breaking abstract • P3 data • Glomerulonephritis • IgA Nephropathy • Renal Disease

Indirect treatment comparison of iptacopan versus pegcetacoplan for patients with paroxysmal nocturnal hemoglobinuria and residual anemia despite C5 inhibitor treatment. (PubMed, J Comp Eff Res) - "No significant difference was noted in CfB in LDH and FACIT-Fatigue score. These findings suggest that iptacopan may improve Hb levels and reduce transfusion dependency compared with pegcetacoplan in PNH patients with residual anemia despite C5i and must be interpreted in the context of ITCs."

Journal • Anemia • Complement-mediated Rare Disorders • Fatigue • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

IgA Nephropathy Insights From Treatment Experience Among Patients Receiving Iptacopan and/or Atrasentan Using Primary Data Collection (clinicaltrials.gov) - P=N/A | N=80 | Not yet recruiting | Sponsor: Novartis Pharmaceuticals

New trial • Glomerulonephritis • IgA Nephropathy • Renal Disease

Efficacy analysis of iptacopan in a patient with thrombotic microangiopathy after allogeneic hematopoietic stem cell transplantation: a case report. (PubMed, Front Immunol) - "The patient had an initial partial response to the C5 inhibitor eculizumab, but the disease progressed...The patient received a total of six sessions of therapeutic plasma exchange and two concurrent doses of defibrotide during the Iptacopan course...This biochemical improvement coincided with key clinical outcomes: resolution of proteinuria and the achievement of sustained red blood cell transfusion independence after day +58 and platelet transfusion independence after day +66, marking a decisive turnaround in his TA-TMA course. Treatment with the novel oral complement inhibitor Iptacopan induced significant hematological and clinical responses in this TA-TMA patient, demonstrating its potential therapeutic efficacy and warranting further clinical investigation."

Journal • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Renal Disease • Transplantation • Transplantation Associated Thrombotic Microangiopathy • IDH1 • STAG2

Low Risk for Meningococcal Infections with Systemically Administered Pegcetacoplan, a Complement C3 and C3b Inhibitor (THSNA 2026) - P3 | "Clinical benefits of the initially available C5 inhibitors that block terminal complement activation (eculizumab, ravulizumab, crovalimab) paved the way for the development of proximal inhibitors, including the C3/C3b inhibitor pegcetacoplan, the factor B inhibitor iptacopan, and the add-on (to C5 inhibitors) factor D inhibitor danicopan. Understanding the safety profile of pegcetacoplan and other complement-targeted therapies will help physicians and patients make informed treatment decisions for individuals with complement-mediated conditions. For over 7 years of total systemic pegcetacoplan exposure, no cases of encapsulated N. meningitidis and consistently low rates of infections by other encapsulated bacteria were observed in patients with PNH, C3G, primary IC-MPGN, or other conditions. These findings may reflect effective risk mitigation strategies."

Complement-mediated Rare Disorders • Glomerulonephritis • Hematological Disorders • Immunology • Infectious Disease • Influenza • Lupus Nephritis • Meningococcal Infections • Nephrology • Paroxysmal Nocturnal Hemoglobinuria • Pneumococcal Infections • Pneumonia • Primary Immunodeficiency • Rare Diseases • Respiratory Diseases • Septic Shock

Pediatric Complement 3 Glomerulonephritis (C3GN) Responsive to Mycophenolate mofetil (NKF-SCM 2026) - "Prednisone (2 mg/Kg/day) and Lisinopril were started at 1.5 months...As UPCR remains elevated (~4.09 mg/mg), empagliflozin was added...RESULTS/CASE DISCUSSION Despite the availability of Pegcetacoplan or Iptacopan, these are not currently approved for children under 12 years of age. Complement inhibitors including Eculizumab/ Ravuluzimab will carry potential life threatening risk of infections. CONCLUSION This case highlights MMF as a viable long-term management strategy in refractory C3GN in young patients intolerant or resistant to corticosteroids."

Clinical • Glomerulonephritis • Infectious Disease • Lupus Nephritis • Nephrology • Pediatrics • Renal Disease

Comparative Network Meta-Analysis (NMA) of Sibeprenlimab Efficacy in the Treatment of Immunoglobulin A Nephropathy (IgAN) (NKF-SCM 2026) - "Treatments analyzed were: sibeprenlimab, atacicept, atrasentan, sparsentan, cemdisiran, TRF-budesonide, ravulizumab, and iptacopan. CONCLUSION Sibeprenlimab demonstrates clinically significant reductions in uPCR compared with other treatments, suggesting potential in IgAN. Updated NMA will be conducted when full Phase 3 results for sibeprenlimab are available."

Retrospective data • Glomerulonephritis • IgA Nephropathy • Renal Disease

ECONOMIC EVALUATION OF PEGCETACOPLAN FOR PNH FROM THE BRAZILIAN PUBLIC HEALTHCARE SYSTEM PERSPECTIVE (ISPOR 2026) - "Comparators included eculizumab, ravulizumab, and iptacopan. From the Brazilian Public Healthcare System perspective, pegcetacoplan represents a cost-saving therapeutic strategy for PNH, yielding lower long-term treatment costs than available C5 inhibitors and offering meaningful reduction in projected national expenditures. These findings support pegcetacoplan as an economically advantageous option for managing previously treated PNH patients."

HEOR • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Primary membranoproliferative glomerulonephritis: natural history, pathogenesis, and treatment. (PubMed, Front Nephrol) - "Two agents are now FDA approved for C3G, the oral Factor B inhibitor iptacopan and the subcutaneous C3-inhibitor pegcetacoplan, the latter also approved for IC-MPGN. If complement inhibition is unavailable, MMF/steroids may be considered. Following transplantation, protocol biopsies are needed to detect early recurrence with the intent of complement inhibition."

Journal • Review • Complement-mediated Rare Disorders • Glomerulonephritis • Immunology • Infectious Disease • Nephrology • Oncology • Transplantation

Overview of Characteristics and Treatment Patterns of US Patients (Pts) With Immunoglobulin A Nephropathy (IgAN) Prescribed Atrasentan: APPRISE-IgAN Data Platform (NKF-SCM 2026) - "INTRODUCTION The A Pt Platform for Real-world data on Iptacopan and Atrasentan in the United StatEs for pts with IgAN (APPRISE-IgAN) data platform captures real-world data on US pts with IgAN prescribed atrasentan (highly selective endothelin A receptor antagonist; accelerated FDA approval Apr 2025) and/or iptacopan (complement pathway factor B inhibitor; accelerated FDA approval Aug 2024). CONCLUSION This analysis highlights characteristics and treatment patterns of pts with IgAN receiving atrasentan. APPRISE-IgAN recruitment and data collection are ongoing and will provide further valuable insights into this population over time."

Clinical • Glomerulonephritis • IgA Nephropathy • Renal Disease