An Open-label, Multicenter Study to Assess the Effect of Zigakibart Treatment on Histologic, Circulating, and Excreted Markers of Kidney Disease and Dysfunction in Adult Patients With IgA Nephropathy.

The purpose of the study is to assess the effect of zigakibart on IgA nephropathy (IgAN) disease progression.

Start Date18 Nov 2025

Sponsor / Collaborator

Novartis Pharmaceuticals Canada, Inc.

NCT06858319

/ RecruitingPhase 3

A Multicenter Open-label Extension Study to Evaluate the Long-term Safety and Tolerability of Zigakibart in Adults With Primary IgA Nephropathy.

The purpose of this study is to determine if zigakibart is safe and effective for long-term use in patients with immunoglobulin A nephropathy (IgAN). This is an extension study for patients who have already completed an another zigakibart study.

Start Date28 Jul 2025

Sponsor / Collaborator

Novartis Pharmaceuticals Canada, Inc.

ChiCTR2500096892

/ RecruitingPhase 3IIT

A Phase 3, Randomized, Double-blind, Placebo-controlled Study of BION-1301 in Adults with IgA Nephropathy(The BEYOND Study)

Start Date25 Sep 2024

Sponsor / Collaborator-

100 Clinical Results associated with Zigakibart

100 Translational Medicine associated with Zigakibart

100 Patents (Medical) associated with Zigakibart

Literatures (Medical) associated with Zigakibart

01 Sep 2025·KIDNEY INTERNATIONAL

Zigakibart demonstrates clinical safety and efficacy in a Phase 1/2 trial of healthy volunteers and patients with IgA nephropathy

Article

Author: Dulos, John ; Kooienga, Laura ; Agha, Irfan ; Smith, William ; van Eenennaam, Hans ; Song, Yuanbo ; Van Elsas, Andrea ; Lo, Jeannette ; Kim, Sung Gyun ; Barratt, Jonathan ; Lee, Eun Young ; Workeneh, Biruh ; Thomas, Hannah

INTRODUCTION:

Zigakibart is a humanized IgG4 monoclonal antibody that binds the cytokine A Proliferation-Inducing Ligand (APRIL, also known as TNFSF13). APRIL is a critical factor in immunoglobulin (Ig) A nephropathy (IgAN) pathogenesis.

METHODS:

Here, we report healthy volunteers (63 overall) and 100-week data from an ongoing Phase 1/2 clinical trial in 40 patients with IgAN (NCT03945318) treated with zigakibart.

RESULTS:

In healthy volunteers, zigakibart was well tolerated following intravenous administration of single doses ranging from 10-1350 mg or multiple doses ranging from 50-450 mg every two weeks. Zigakibart exposure increased in a dose-proportional manner, with corresponding durable reductions in levels of free APRIL, IgA and IgM, and to a lesser extent, IgG. In patients with IgAN, zigakibart 600 mg, administered subcutaneously every two weeks, was well tolerated with no treatment-emergent adverse events leading to study drug discontinuation or death. A 60% reduction in proteinuria and sustained estimated glomerular filtration rate stabilization were observed at week 100. There was a notable decrease in hematuria, as well as rapid and durable reductions in IgA, galactose-deficient IgA (Gd-IgA1), and IgM levels, with a modest reduction in IgG.

CONCLUSIONS:

Overall, zigakibart demonstrated robust pharmacological activity, and clinical evidence shows an acceptable safety profile with clinically meaningful proteinuria reduction and sustained estimated glomerular filtration rate stabilization in patients with IgAN, providing a potentially disease-modifying approach for the treatment of IgAN. The effects of zigakibart on proteinuria and long-term kidney function in adults with IgAN are being evaluated in the ongoing phase 3 BEYOND study (NCT05852938).

04 Dec 2023·Clinical kidney journal

The contribution of a proliferation-inducing ligand (APRIL) and other TNF superfamily members in pathogenesis and progression of IgA nephropathy

Review

Author: Barratt, Jonathan ; Yeo, See Cheng

ABSTRACT:

Advances in our understanding of the pathogenesis of immunoglobulin A nephropathy (IgAN) have led to the identification of novel therapeutic targets and potential disease-specific treatments. Specifically, a proliferation-inducing ligand (APRIL) has been implicated in the pathogenesis of IgAN, mediating B-cell dysregulation and overproduction of pathogenic galactose-deficient IgA1 (Gd-IgA1). Animal and clinical studies support the involvement of APRIL in the pathogenesis and progression of IgAN. An elevated level of APRIL is found in IgAN when compared with controls, which correlates with the level of Gd-IgA1 and associates with more severe disease presentation and worse outcomes. Conversely, anti-APRIL therapy reduces pathogenic Gd-IgA1 and IgA immune complex formation and ameliorates the severity of kidney inflammation and injury. Genome-wide association studies in IgAN have identified TNFSF13 and TNFRSF13B, a cytokine ligand-receptor gene pair encoding APRIL and its receptor, respectively, as risk susceptibility loci in IgAN, further supporting the causal role of the APRIL signalling pathway in IgAN. Several novel experimental agents targeting APRIL, including atacicept, telitacicept, zigakibart and sibeprenlimab, are currently under investigation as potential therapies in IgAN. Preliminary results suggest that these agents are well-tolerated, and reduce levels of Gd-IgA1, with corresponding improvement in proteinuria. Further studies are ongoing to confirm the safety and efficacy of anti-APRIL approaches as an effective therapeutic strategy in IgAN.

01 Dec 2023·Clinical rheumatology

A narrative review of potential drug treatments for nephritis in children with IgA vasculitis (HSP)

Review

Author: Lamond, Megan ; Chetwynd, Andrew J ; Marro, Julien ; Oni, Louise ; Williams, Chloe E C

Abstract:

Immunoglobulin A (IgA) vasculitis (IgAV, also known as Henoch-Schoenlein purpura, HSP) is the most common vasculitis of childhood. It usually presents with a simple, self-limiting disease course; however, a small subset of patients may develop kidney involvement (IgAV-N) which occurs 4–12 weeks after disease onset and is the biggest contributor to long-term morbidity. Treatment currently targets patients with established kidney involvement; however; there is a desire to work towards early prevention of inflammation during the window of opportunity between disease presentation and onset of significant nephritis. There are no clinical trials evaluating drugs which may prevent or halt the progression of nephritis in children with IgAV apart from the early use of corticosteroids which have no benefit. This article summarises the latest scientific evidence and clinical trials that support potential therapeutic targets for IgAV-N that are currently being developed based on the evolving understanding of the pathophysiology of IgAV-N. These span the mucosal immunity, B-cell and T-cell modulation, RAAS inhibition, and regulation of complement pathways, amongst others. Novel drugs that may be considered for use in early nephritis include TRF-budesonide; B-cell inhibiting agents including belimumab, telitacicept, blisibimod, VIS649, and BION-1301; B-cell depleting agents such as rituximab, ofatumumab, and bortezomib; sparsentan; angiotensin converting enzyme inhibitors (ACE-Is); and complement pathway inhibitors including avacopan, iptacopan, and narsoplimab. Further clinical trials, as well as pre-clinical scientific studies, are needed to identify mechanistic pathways as there may be an opportunity to prevent nephritis in this condition.Key Points• Kidney involvement is the main cause of long-term morbidity and mortality in IgA vasculitis despite the current treatment recommendations.• The evolving understanding of the pathophysiology of IgA vasculitis is allowing exploration of novel treatment options which target underlying immune pathways.• Novel treatments currently being trialled in IgA nephropathy may have benefit in IgA vasculitis due to the similarities in the underlying pathophysiology, such as TRF-budesonide, B-cell modulators, and complement inhibitors.• Further studies, including clinical trials of novel drugs, are urgently needed to improve the long-term outcomes for children with IgA vasculitis nephritis.

141

News (Medical) associated with Zigakibart

26 Nov 2025

·www.fiercepharma.com

In crowded kidney disease space, Otsuka receives FDA approval for first-in-class Voyxact

Vertex, Novartis and Vor Bio are developing anti-APRIL drugs against IgAN, too. A new FDA approval for a first-in-class drug has vaulted Otsuka into immunoglobulin A nephropathy (IgAN), an increasingly competitive kidney disease field targeted by multiple biopharma companies.The new drug, called Voyxact, has won an FDA accelerated approval to be used to reduce proteinuria in adults with primary IgAN who are at risk of progression, Otsuka said Tuesday.A self-administered injection given once per month, the drug works by blocking A proliferation-inducing ligand (APRIL), a protein that promotes autoantibody production that may trigger inflammation and kidney damage, causing IgAN.The drug, also known as sibeprenlimab, is the first in a growing list of candidates to reach the market targeting this pathway. Other drugmakers, including Vertex, Novartis and Vor Bio are working on anti-APRIL agents against IgAN and other disorders. Voyxact is also the first biologic drug approved for IgAN, whereas all four earlier IgAN FDA approvals went to small molecules.Voyxact won the FDA’s endorsement by proving its ability to reduce proteinuria, or protein in the urine, as measured by the urinary protein-to-creatinine ratio (UPCR). In a phase 3 trial coded Visionary, Voyxact led to a significant placebo-adjusted 51% reduction in proteinuria at nine months of treatment. Patients enrolled in the trial were receiving standard therapies such as ACE inhibitors, angiotensin II receptor blockers and/or SGLT2 inhibitors.After one year of treatment, Voyxact’s placebo-adjusted treatment effect on proteinuria reached 54.3%, according to results presented a few days ago at the American Society of Nephrology Kidney Week 2025 and published in The New England Journal of Medicine.“The availability of Voyxact represents a novel targeted approach to help manage this complex disease for patients living with IgAN,” John Kraus, M.D., Ph.D., chief medical officer of Otsuka, said in a Nov. 25 release. “With its targeted mechanism, strong efficacy, safety profile, and once-every-four-weeks dosing, Voyxact offers a new option for IgAN patients.”Unlike previous accelerated approvals in IgAN, the FDA is not restricting the patient population eligible for Voyxact based on a specific UPCR level. Novartis’ endothelin A receptor antagonist (ERA) Vanrafia, the most recent accelerated approval in IgAN doled out by the FDA in April, is currently allowed for patients with UPCR of at least 1.5 g/g. Travere Therapeutics’ dual ARB/ERA drug Filspari shook off that same UPCR limitation only after winning a full approval last year.To win a traditional nod, Otsuka is on the hook to show whether Voyxact can slow disease progression, as measured by estimated glomerular filtration rate (eGFR) decline, at two years in the ongoing Visionary study. Otsuka said it expects to have that data in early 2026.“We think FDA is comfortable with the eGFR trajectory of this class of drugs to allow broad use,” Jefferies analysts said of Voyxact’s label in a Nov. 25 note.The FDA’s decision here bodes well for future contenders in the APRIL class, the Jefferies team observed.According to Jefferies’ calculations, IgAN represents a large market worth around $6 billion to $10 billion in potential sales per year. But competition within the APRIL class will be intense, the analysts said, and the space is poised to get crowded fast.Several companies are developing BAFF/APRIL duo inhibitors. BAFF, or B-cell activating factor, is another key mediator in the production of abnormal proteins and autoantibodies involved in IgAN.Vera Therapeutics announced on Nov. 7 that it has submitted an FDA application for its BAFF/APRIL med, atacicept, which yielded a 42% reduction in UPCR compared with placebo at nine months at an interim analysis of the phase 3 Origin 3 trial.Through its $4.9 billion acquisition of Alpine Immune Sciences last year, Vertex is preparing an FDA application for an accelerated approval for its BAFF/APRIL drug, povetacicept, in IgAN. The company has said it will begin an FDA rolling submission this year and intends to use a priority review voucher to expedite the regulatory process, potentially leading to a go-ahead by the end of 2026.In the phase 2 Ruby-3 trial, povetacicept elicited absolute proteinuria reductions of 64% at 48 weeks, according to data presented at the recent ASN annual meeting. The trial does not have a placebo control arm.Meanwhile, Vor Bio’s partner RemeGen just reported positive China phase 3 data for its BAFF/APRIL duo inhibitor telitacicept in IgAN, although the drug’s U.S. clinical development plan in the disease remains unclear. What’s more, Novartis has an anti-APRIL drug called zigakibart in addition to FDA-approved Vanrafia and factor B inhibitor Fabhalta, which the company said has shown it can slow kidney function decline and IgAN progression in the final analysis of a phase 3 trial.Otsuka has some experience in the kidney disease space with Jynarque, which was approved by the FDA in 2018 as the first treatment to slow kidney function decline in patients with autosomal dominant polycystic kidney disease (ADPKD).

Clinical ResultDrug ApprovalPhase 3AcquisitionPhase 2

04 Nov 2025

·www.globenewswire.com

New Novartis data at ASN Kidney Week and AHA Scientific Sessions demonstrate momentum of broad CRM portfolio and pipeline

Fabhalta and Vanrafia Phase III analyses, Fabhalta real-world treatment pattern data, and zigakibart Phase I/II trial showcase strength of IgA nephropathy (IgAN) portfolioC3 glomerulopathy (C3G) studies add to body of evidence of Fabhalta long-term safety and efficacy profile in native and post-transplant disease recurrence patients V-INCEPTION analyses highlight Leqvio adherence and goal attainment data when initiated early in post-acute coronary syndrome patientsLp(a) data underscore importance of early detection of this independent CVD risk factor in premature ASCVD patients and potential benefit of Lp(a)-targeted therapies Basel, November 4, 2025 – Novartis will present new data from 33 abstracts across its Cardiovascular, Renal, and Metabolic (CRM) disease portfolio at the upcoming American Society of Nephrology (ASN) Kidney Week 2025 in Houston, Texas and American Heart Association's (AHA) Scientific Sessions 2025 in New Orleans, Louisiana, advancing scientific insight into these critical disease areas.“Novartis will present a broad range of data at ASN and AHA demonstrating innovation and commitment to advancing therapies that address root causes of heart and kidney conditions,” said Ruchira Glaser, M.D., Global Head, Cardiovascular, Renal and Metabolic Development Unit, Novartis. "Studies on both our approved and investigational therapies in these closely connected therapeutic areas reflect our longstanding dedication to deliver meaningful solutions for patients living with some of the rarest to most prevalent CRM diseases worldwide.”Key highlights of data accepted by ASN include: Abstract Title Abstract Number/Presentation Details Fabhalta® (iptacopan) Efficacy and Safety of Iptacopan in Patients (Pts) from East Asia with IgAN: Interim Results from the Phase 3 APPLAUSE-IgAN Trial Abstract #PO0811Poster PresentationNovember 7, 10:00 AM – 12:00 PM CT Early Insights into Characteristics and Treatment Patterns of US Patients (Pts) with IgAN Who Were Prescribed Iptacopan: The APPRISE-IgAN Data Platform Abstract #PO0771Poster PresentationNovember 8, 10:00 AM – 12:00 PM CT Efficacy and Safety of Iptacopan in Patients with C3 Glomerulopathy: C3G Extension Trial Interim Results from the Phase 3 APPEAR-C3G Patients Abstract #PO0838Poster PresentationNovember 7, 10:00 AM – 12:00 PM CT Update to the Long-Term Efficacy and Safety of Iptacopan in C3 Glomerulopathy: 39-Month Phase 2 Extension Study Data Abstract #PO0837Poster PresentationNovember 7, 10:00 AM – 12:00 PM CT Iptacopan Treatment in Patients with C3 Glomerulopathy (C3G): 12-Month Results from the Early Access Program Abstract #PO0811Poster PresentationNovember 8, 10:00 AM – 12:00 PM CT Vanrafia® (atrasentan) Renin-Angiotensin System Inhibitor (RASi) Use and Atrasentan in IgAN: Post Hoc Analysis from the ALIGN Trial Abstract #PO0827Poster PresentationNovember 7, 10:00 AM – 12:00 PM CT Efficacy and Safety of Atrasentan in Patients (Pts) with IgAN from East (E) Asia: Phase 3 ALIGN Interim Data Abstract #OR034Oral PresentationNovember 7, 5:30 – 5:40 PM CT Zigakibart Long-Term Stabilization of Kidney Function Regardless of Baseline eGFR and Urine Protein-to-Creatinine Ratio (UPCR) in Patients with IgAN: Subgroup Analyses from the Phase 1/2 Trial of Zigakibart Abstract #PO0814Poster PresentationNovember 7, 10:00 AM – 12:00 PM CT SHIFT: A Kidney Biopsy Study in Adults with IgAN Treated with Zigakibart Abstract #INFO18Informational PosterNovember 7, 10:00 AM – 12:00 PM CT Farabursen Farabursen Increases Urinary Polycystin-1 and Polycystin-2 and Reduces Height-Adjusted Total Kidney Volume Growth in Patients with ADPKD Abstract #OR089Oral PresentationNovember 8, 4:30 PM – 6:00 PM CT Key highlights of data accepted by AHA include: Abstract Title Abstract Number/Presentation Details Leqvio® (inclisiran)* Effect Of Inclisiran-based Treatment Strategy, In Combination With Individually Optimized Statin Therapy, On Quality Of Life And Muscle-related Pain vs. Standard of Care: Exploratory Outcomes From The VICTORION-Difference Study Abstract #MP1723Moderated Digital Poster PresentationNovember 9, 12:25 PM – 12:30 PM CT VICTORION-INCEPTION: Adherence and Goal Attainment Data Support the Addition of Inclisiran to Background Lipid-Lowering Therapy as a Lipid Management Strategy Post-Acute Coronary Syndrome Abstract #MP1492Moderated Digital Poster PresentationNovember 9, 10:18 AM – 10:23 AM CT VICTORION-INCEPTION: Consistent Low-Density Lipoprotein Cholesterol Lowering With Inclisiran Following Acute Coronary Syndrome Independent of Index Lipid-Lowering Therapy Abstract #Mo2111Poster Presentation November 10, 1:00 PM – 2:00 PM CT Primary Results of the VICTORION-NOVEL (LDL-C maNagement PrOgram in Atherosclerotic Cardiovascular Disease (ASCVD) Patients with Elevated LDL-C) Lipid Optimization Multicenter Implementation Trial Abstract #MP2380Moderated Digital Poster PresentationNovember 10, 1:59 PM – 2:04 PM CT Lipoprotein(a) Overcoming Barriers For Research Participation In Minority Patients In Lp(a)FRONTIERS EXPANSION: A Randomized, Double-Blind, Placebo-Controlled, Multicenter Study To Evaluate Efficacy And Safety Of Pelacarsen In U.S. Black And Hispanic Patients with Elevated Lp(a) And Established ASCVD Abstract #MP913Moderated Digital Poster PresentationNovember 8, 1:45 PM – 1:50 PM CT Elevated Lipoprotein(a) Is Independently Associated With Greater Infarct Size, Especially in Premature Atherosclerosis Abstract #MP376Moderated Digital Poster PresentationNovember 8, 9:50 AM – 9:55 AM CT Impact of Elevated Lipoprotein(a) on Cardiovascular Events in Patients with premature ASCVD: A Nationally Representative Sample of US Medicare, Medicaid, and Commercial Enrollees Abstract #MP2201Moderated Digital Poster PresentationNovember 10, 2:20 PM – 2:25 PM CT Pacibekitug** A Phase 2 Multicenter, Randomized, Double-Blind, Placebo-Controlled Study of Monthly or Quarterly Subcutaneous Administration of the Interleukin-6 Inhibitor Pacibekitug in Patients With Elevated High-Sensitivity C-Reactive Protein and Chronic Kidney Disease: 90-Day Analyses from TRANQUILITY Abstract # MP1719Moderated Digital Poster PresentationNovember 9, 11:57 AM – 12:02 PM CT Product InformationFor full prescribing information, including approved indications and important safety information about marketed products, please visit https://www.novartis.com/about/products.Novartis in cardiovascular, renal, and metabolic diseaseNovartis is redefining how the world tackles cardiovascular, renal, and metabolic (CRM) conditions because interconnected diseases require interconnected thinking. We are continuing our 40-year legacy in both cardiovascular (CV) and renal with our bold science, robust portfolio, and growing pipeline. In cardiovascular care, we envision a world with no preventable CV deaths and are using cutting-edge science and technology in our mission to ensure no heart is lost too soon. In kidney disease, we are building on our groundbreaking work that began in transplant, with an expanding portfolio of medicines that target the underlying causes of disease, starting with kidney conditions that have significant unmet need. Together, we are breaking boundaries – to transform outcomes, improve lives, and build a better world for people with CRM diseases.DisclaimerThis press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as “potential,” “can,” “will,” “plan,” “may,” “could,” “would,” “expect,” “anticipate,” “look forward,” “believe,” “committed,” “investigational,” “pipeline,” “launch,” or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for the investigational or approved products described in this press release, or regarding potential future revenues from such products. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that such products will be commercially successful in the future. In particular, our expectations regarding such products could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AG’s current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.About Novartis Novartis is an innovative medicines company. Every day, we work to reimagine medicine to improve and extend people’s lives so that patients, healthcare professionals and societies are empowered in the face of serious disease. Our medicines reach nearly 300 million people worldwide.Reimagine medicine with us: Visit us at https://www.novartis.com and connect with us on LinkedIn, Facebook, X/Twitter and Instagram.*Novartis has obtained global rights to develop, manufacture and commercialize Leqvio under a license and collaboration agreement with Alnylam Pharmaceuticals, a leader in RNAi therapeutics.**Complementing its cardiovascular pipeline, Novartis recently acquired Tourmaline Bio, a clinical-stage biopharmaceutical company developing pacibekitug, a Phase III-ready anti-IL-6monoclonal antibody for atherosclerotic cardiovascular disease (ASCVD). # # # Novartis Media RelationsE-mail: media.relations@novartis.com Novartis Investor RelationsCentral investor relations line: +41 61 324 7944E-mail: investor.relations@novartis.com

Phase 3Phase 2License out/inAHAClinical Result

17 Oct 2025

·www.globenewswire.com

Novartis Fabhalta® (iptacopan) meets Phase III primary endpoint, slows kidney function decline in patients with IgA nephropathy (IgAN)

In APPLAUSE-IgAN final analysis, Fabhalta demonstrated statistically significant, clinically meaningful improvement in estimated glomerular filtration rate (eGFR) slope vs. placebo over two years1 eGFR is key marker of kidney function; IgAN is progressive autoimmune kidney disease that leads to kidney failure in many patients1-3 Fabhalta is first and only approved complement inhibitor for adults with IgAN and has potential to delay disease progression4,5 Fabhalta received accelerated approval for reduction of proteinuria in adults with IgAN in US in 2024; data support 2026 submission for traditional FDA approval4,5 October 16, 2025 – Novartis today announced positive final results from APPLAUSE-IgAN, a Phase III study evaluating Fabhalta® (iptacopan) in adults living with IgA nephropathy (IgAN). Fabhalta, an oral alternative complement pathway inhibitor, demonstrated statistically significant, clinically meaningful superiority compared to placebo in slowing IgAN progression measured by annualized total slope of estimated glomerular filtration rate (eGFR) decline over two years1. “Progressive diseases such as IgAN present an urgent need for interventions that can ultimately improve kidney health. Many people with IgAN commonly experience fear and anxiety of disease progression,” said Ruchira Glaser, Development Unit Head, Cardiovascular, Renal & Metabolic, Novartis. “We are excited about today’s positive Phase III APPLAUSE-IgAN results showing slowed eGFR decline, which add to the growing evidence of Fabhalta as a targeted therapy to preserve long-term kidney function, giving hope to people living with this condition.” Novartis intends to use these data to support Fabhalta submissions in 2026. Alongside Fabhalta, Novartis continues to advance its multi-asset IgAN portfolio, which also includes Vanrafia® (atrasentan) and investigational compound zigakibart. IgAN is a progressive autoimmune kidney disease with approximately 25 per million people newly diagnosed worldwide each year3. IgAN is highly debilitating as it leads to glomerular inflammation, proteinuria, and a gradual decline in eGFR2. Up to 50% of patients with persistent proteinuria progress to kidney failure within 10 to 20 years of diagnosis, often requiring dialysis or kidney transplantation as part of long-term disease management2,6,7. Furthermore, people living with IgAN often face mental, social, and economic challenges2,8. Supportive care does not address the underlying causes of the disease and often fails to slow disease progression, reinforcing the need for more targeted therapies for IgAN3,9. In APPLAUSE-IgAN, Fabhalta was well tolerated with a favorable safety profile in line with previously reported data10. Full data from the APPLAUSE-IgAN final analysis will be presented at future medical meetings. Fabhalta (iptacopan) is an oral, Factor B inhibitor of the alternative complement pathway10. Discovered at Novartis, Fabhalta received US Food and Drug Administration (FDA) and European Commission (EC) approval in December 2023 and May 2024, respectively, for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH). Fabhalta also received accelerated approval in the US in August 2024, and in China in September 2025, for the reduction of proteinuria in adults with primary IgA nephropathy (IgAN) at risk of rapid disease progression (generally UPCR ≥1.5 g/g4,5,11,12. In 2025, Fabhalta received FDA and EC approval as well as approvals in China and Japan for the treatment of adults with C3 glomerulopathy (C3G), making it the first treatment approved for this condition13-15. Fabhalta is being studied in a broad range of rare kidney diseases, including atypical hemolytic uremic syndrome (aHUS), immune complex membranoproliferative glomerulonephritis (IC-MPGN) and lupus nephritis (LN)16-18. Studies are ongoing to evaluate the safety and efficacy profiles in these investigational indications and support potential regulatory submissions16-18. APPLAUSE-IgAN (NCT04578834) is a Phase III multicenter, randomized, double-blind, placebo-controlled, parallel-group study to evaluate the efficacy and safety of twice-daily oral Fabhalta (200 mg) in 477 adult primary IgAN patients (main study population). Patients were randomized to Fabhalta or placebo, on top of supportive care (a stable dose of maximally-tolerated renin-angiotensin system (RAS) inhibitor therapy with or without a stable dose of SGLT2i)1. The two primary endpoints of the study for the interim and final analysis, respectively, are proteinuria reduction at 9 months as measured by UPCR, and the annualized total eGFR slope over 24 months10,18. During the final analysis, the following secondary endpoints were assessed: proportion of participants reaching UPCR 2 or maintenance dialysis or receipt of kidney transplant or death from kidney failure), and change from baseline to 9 months in the fatigue scale measured by the Functional Assessment Of Chronic Illness Therapy-Fatigue questionnaire19. The main study population enrolled patients with an eGFR ≥30 mL/min/1.73 m2 and UPCR ≥1 g/g at baseline10,18. In addition, a smaller cohort of patients with severe renal impairment (eGFR 20–30 mL/min/1.73 m2 at baseline) was also enrolled to provide additional information but not contributing to the main efficacy analyses10,19. Novartis is an innovative medicines company. Every day, we work to reimagine medicine to improve and extend people’s lives so that patients, healthcare professionals and societies are empowered in the face of serious disease. Our medicines reach nearly 300 million people worldwide. References Novartis. Data on file. Kwon CS, Daniele P, Forsythe A et al. A systematic literature review of the epidemiology, health-related quality of life impact, and economic burden of immunoglobulin a nephropathy. J Health Econ Outcomes Res 2021;8:36–45. Cheung C, Barratt J. The rapidly changing treatment landscape of IgA nephropathy. Semin Nephrol 2025;44:151573. Novartis Pharmaceuticals Corporation. Fabhalta prescribing information. 2023 (2025 update). Available at: https://www.novartis.com/us-en/sites/novartis_us/files/fabhalta.pdf (accessed October 2025). Novartis Pharmaceuticals Corporation. Novartis receives FDA accelerated approval for Fabhalta® (iptacopan), the first and only complement inhibitor for the reduction of proteinuria in primary IgA nephropathy (IgAN) (2024). Available at: https://www.novartis.com/news/media-releases/novartis-receives-fda-accelerated-approval-fabhalta-iptacopan-first-and-only-complement-inhibitor-reduction-proteinuria-primary-iga-nephropathy-igan (accessed October 2025). Pitcher D, Braddon F, Hendry B et al. Long-term outcomes in IgAN. Clin J Am Soc Nephrol 2023;18:727–8. Mohd R, Mohammad Kazmin NE, Abdul Cader R, et al. Long-term outcome of immunoglobulin A (IgA) nephropathy: a single center experience. PLoS One. 2021;16:e0249592. National Kidney Foundation. The voice of the patient (2020). Available at: https://igan.org/wp-content/uploads/2021/01/VOP_IgAN_12-7-20__FNL.pdf (accessed October 2025). Kidney Disease: Improving Global Outcomes (KDIGO) Glomerular Diseases Work Group. KDIGO 2021 Clinical Practice Guideline for the Management of Glomerular Diseases. Kidney Int 2021;100:S1–276. Perkovic V, Barratt J, Rovin B, et al. Alternative complement pathway inhibition with iptacopan in IgA nephropathy. N Engl J Med. 2025;392:531–543. Fabhalta®. EMA Summary of Product Characteristics. Novartis Europharm Limited (2024). Available at: https://www.ema.europa.eu/en/documents/product-information/fabhalta-epar-product-information_en.pdf (accessed October 2025). PHARMCUBE. Novartis expands indications for iptacopan, capmatinib in China (2025). Available at: Novartis Expands Indications for Iptacopan, Capmatinib in China PharmCube-News & Reports (accessed October 2025). Fineline Cube. Novartis’ fabhalta approved in China for C3 glomerulopathy (2025). Available at: Novartis' Fabhalta Approved in China for C3 Glomerulopathy - Insight, China's Pharmaceutical Industry (accessed October 2025). Pharma Japan. Novartis makes full foray into nephrology space with fabhalta: exec (2025). Available at: Novartis Makes Full Foray into Nephrology Space with Fabhalta: Exec | PHARMA JAPAN (accessed October 2025). Novartis. Press release. Novartis receives third FDA approval for oral Fabhalta® (iptacopan) – the first and only treatment approved in C3 glomerulopathy (C3G) (2025). Available at: https://www.novartis.com/news/media-releases/novartis-receives-third-fda-approval-oral-fabhalta-iptacopan-first-and-only-treatment-approved-c3-glomerulopathy-c3g (accessed October 2025). Clinicaltrials.gov. NCT04889430. Efficacy and Safety of Iptacopan (LNP023) in Adult Patients with Atypical Hemolytic Uremic Syndrome Naive to Complement Inhibitor Therapy (APPELHUS). Available at: https://clinicaltrials.gov/study/NCT04889430 (accessed October 2025). Clinicaltrials.gov. NCT05755386. Study of Efficacy and Safety of Iptacopan in Participants With IC-MPGN (APPARENT). Available at: https://clinicaltrials.gov/study/NCT05755386 (accessed October 2025). Clinicaltrials.gov. NCT05268289. Study of Efficacy and Safety of LNP023 in Participants With Active Lupus Nephritis Class III-IV, +/ - V. Available at: https://clinicaltrials.gov/study/NCT05268289 (accessed October 2025). Rizk DV, Rovin BH, Zhang H et al. Targeting the alternative complement pathway with iptacopan to treat IgAN: design and rationale of the APPLAUSE-IgAN study. Kidney Int Rep 2023;8:968–79. The content above comes from the network. if any infringement, please contact us to modify.

Clinical ResultDrug Approval

100 Deals associated with Zigakibart

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Glomerulonephritis, IGA	Phase 3	China	Novartis Pharmaceuticals Canada, Inc.	06 Jul 2023
Glomerulonephritis, IGA	Phase 3	Japan	Novartis Pharmaceuticals Canada, Inc.	06 Jul 2023
Glomerulonephritis, IGA	Phase 3	Argentina	Novartis Pharmaceuticals Canada, Inc.	06 Jul 2023
Glomerulonephritis, IGA	Phase 3	Australia	Novartis Pharmaceuticals Canada, Inc.	06 Jul 2023
Glomerulonephritis, IGA	Phase 3	Belgium	Novartis Pharmaceuticals Canada, Inc.	06 Jul 2023
Glomerulonephritis, IGA	Phase 3	Brazil	Novartis Pharmaceuticals Canada, Inc.	06 Jul 2023
Glomerulonephritis, IGA	Phase 3	Canada	Novartis Pharmaceuticals Canada, Inc.	06 Jul 2023
Glomerulonephritis, IGA	Phase 3	Croatia	Novartis Pharmaceuticals Canada, Inc.	06 Jul 2023
Glomerulonephritis, IGA	Phase 3	Czechia	Novartis Pharmaceuticals Canada, Inc.	06 Jul 2023
Glomerulonephritis, IGA	Phase 3	France	Novartis Pharmaceuticals Canada, Inc.	06 Jul 2023

Clinical Result

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


Pubmed \| Kidney Int	Phase 1/2	Glomerulonephritis, IGA APRIL \| IgA \| IgM ... View more	103	Zigakibart 600 mg	nlsqybbdcb(vwwfsmdmby) = In healthy volunteers, zigakibart was well tolerated following intravenous administration of single doses ranging from 10-1350 mg or multiple doses ranging from 50-450 mg every two weeks. Zigakibart exposure increased in a dose-proportional manner, with corresponding durable reductions in levels of free APRIL, IgA and IgM, and to a lesser extent, IgG. In patients with IgAN, zigakibart 600 mg, administered subcutaneously every two weeks, was well tolerated with no treatment-emergent adverse events leading to study drug discontinuation or death. oodvvpyvxd (lfwfblfffo )	Positive	01 Sep 2025
NCT03945318 (Phase 1/2 Study of BION-1301 \| ADU-CL-19, PRNewswire) Manual	Phase 1/2	Glomerulonephritis, IGA	40	Zigakibart	hwgclgntah(cjzqyxzxrs) = adxrluilmd kuyvwcqrii (pmjyhfootc ) View more	Positive	05 Jun 2025
NCT03945318 (ADU-CL-19, ASN2025 \| PRNewswire \| Pubmed)	Phase 1/2	Glomerulonephritis, IGA	40	zigakibart	cfzgivdmvr(twlzpxnpqr) = kmqhafakqf umsvbtdahq (nlpikxbxgr ) View more	Positive	30 May 2025
NCT03945318 (ADU-CL-19, ASN2024)	Phase 1/2	Glomerulonephritis, IGA	40	Zigakibart 450 mg Q2W IV→600 mg Q2W SC	rjtgdtuchy(bbnigealkh) = tfvcfvedqb nvjghtrqev (vxtiswxdgw )	Positive	25 Oct 2024
NCT03945318 (ADU-CL-19, ISN WCN2023)	Phase 1/2	Glomerulonephritis, IGA APRIL \| IgA \| Gd-IgA1 ... View more	-	BION-1301 450 mg IV	zdbqdfmcko(uwkywbzwav) = yjtbzvmray cnzomfbtij (wtpqesyyjy )	Positive	01 Mar 2023
NCT03945318 (ADU-CL-19, ISN WCN2023)	Phase 1/2	Glomerulonephritis, IGA APRIL \| IgA \| Gd-IgA1 ... View more	-	BION-1301 600 mg SC	zdbqdfmcko(uwkywbzwav) = bnkzhdpflj cnzomfbtij (wtpqesyyjy )	Positive	01 Mar 2023
NCT03945318 (Phase 1/2 Study of BION-1301, ASN2022)	Phase 1/2	Glomerulonephritis, IGA APRIL \| IgA \| Gd-IgA1 ... View more	20	BION-1301 450 mg IV	vbehdhibfi(pioitbpfnz) = xpuakfzypm hpvvrqxjzl (ksrpqwskgx )	Positive	04 Nov 2022
NCT03945318 (Phase 1/2 Study of BION-1301, ASN2022)	Phase 1/2	Glomerulonephritis, IGA APRIL \| IgA \| Gd-IgA1 ... View more	20	BION-1301 600 mg SC	vbehdhibfi(pioitbpfnz) = lfvfpcqnzd hpvvrqxjzl (ksrpqwskgx )	Positive	04 Nov 2022
NCT03945318 (Corporate Publications) Manual	Phase 1/2	Glomerulonephritis, IGA	40	BION-1301 (450 mg Q2W IV → 600 mg Q2W SC, up to 104 weeks ‖)	leojocvlmj(pqtqqugnfd) = ovjkjsfjjt hqkalclmvs (yzyxmsjutm ) View more	Positive	01 Nov 2022
NCT03945318 (Corporate Publications) Manual	Phase 1/2	Glomerulonephritis, IGA	40	BION-1301 (600 mg Q2W de novo SC, up to 104 weeks)	leojocvlmj(blwonstzkf) = ganvgkmfkz vxixzvyvof (cipvdfsqsh )	Positive	01 Nov 2022
NCT03945318 (Corporate Publications) Manual	Phase 1/2	Glomerulonephritis, IGA	10	BION-1301	zdikdyjxiz(baxrktrcla) = kgijxxtfga caadfltepo (oenkgszdlm ) View more	Positive	04 Nov 2021
NCT03340883 (CTgov)	Phase 1/2	Relapse multiple myeloma \| Refractory Multiple Myeloma	21	BION-1301 (BION-1301 50 mg Q2W)	pwptolxtvt = rdpgmkwlzc mvbtdlonrc (wujbygadyt, uqtuvmuphd - eauazmmiiz) View more	-	07 Sep 2020
NCT03340883 (CTgov)	Phase 1/2	Relapse multiple myeloma \| Refractory Multiple Myeloma	21	BION-1301 (BION-1301 150 mg Q2W)	pwptolxtvt = mfrdkvzeeb mvbtdlonrc (wujbygadyt, soyyvhvuqv - ubvccxozix) View more	-	07 Sep 2020

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