RegulationPriority Review (US), Breakthrough Therapy (US), Accelerated Approval (US), Orphan Drug (US), Orphan Drug (EU), PRIME (EU), Priority Review (CN), Breakthrough Therapy (CN), Orphan Drug (KR), Orphan Drug (AU), Rare Pediatric Disease (US)

Structure

Molecular FormulaC25H33ClN2O5

InChIKeyJUWBBUFSAGEROP-VVJLZRNGSA-N

CAS Registry2447007-60-3

Clinical Trials associated with Iptacopan

CTR20243185 / RecruitingPhase 3

一项在全身型重症肌无力（gMG）患者中评价iptacopan的疗效、安全性和耐受性的随机、双盲、安慰剂对照III期研究，以及随后的一项开放性扩展研究

[Translation] A randomized, double-blind, placebo-controlled Phase III study evaluating the efficacy, safety, and tolerability of iptacopan in patients with generalized myasthenia gravis (gMG), followed by an open-label extension study

证明在接受稳定SOC治疗的gMG患者中，iptacopan与安慰剂相比在治疗6个月（第180天）时降低重症肌无力日常生活活动（MG-ADL）量表总分方面的疗效

[Translation]

To demonstrate the efficacy of iptacopan compared with placebo in reducing the total score of the Myasthenia Gravis Activities of Daily Living (MG-ADL) scale at 6 months (Day 180) in patients with gMG receiving stable SOC therapy

Start Date21 Nov 2024

Sponsor / Collaborator

Novartis Pharma Produktions GmbH

[+2]

CTR20241663 / RecruitingPhase 3

一项评估aHUS研究受试者从抗C5抗体治疗转换至iptacopan治疗的有效性和安全性的多中心、单臂、开放标签研究

[Translation] A multicenter, single-arm, open-label study to evaluate the efficacy and safety of switching aHUS study subjects from anti-C5 antibody therapy to iptacopan therapy

本研究的目的是评估接受抗C5抗体治疗产生临床应答的成人aHUS研究受试者（年龄 ≥ 18岁）从抗C5抗体治疗（依库珠单抗或ravulizumab）转换至iptacopan治疗的有效性和安全性。本研究将为从关键性III期研究CLNP023F12301获得的iptacopan治疗aHUS受试者的获益/风险关系提供支持性证据（从抗C5抗体治疗转换为iptacopan治疗）。本研究为一项单臂、开放标签研究，期间研究受试者将接受研究药物治疗24个月。主要终点是在iptacopan治疗的前12个月内无血栓性微血管病（TMA）表现的受试者比例。

[Translation]

The purpose of this study is to evaluate the efficacy and safety of switching from anti-C5 antibody therapy (eculizumab or ravulizumab) to iptacopan in adult aHUS study subjects (age ≥ 18 years) who have achieved a clinical response to anti-C5 antibody therapy. This study will provide supportive evidence for the benefit/risk relationship of iptacopan in aHUS subjects (switching from anti-C5 antibody therapy to iptacopan) obtained from the pivotal Phase III study CLNP023F12301. This study is a single-arm, open-label study during which study subjects will receive study medication for 24 months. The primary endpoint is the proportion of subjects who are free of thrombotic microangiopathy (TMA) during the first 12 months of iptacopan treatment.

Start Date11 Sep 2024

Sponsor / Collaborator

Novartis Pharma Produktions GmbH

[+2]

NCT06388941 / RecruitingPhase 2

A Randomized, Controlled Study to Evaluate LNP023 (Iptacopan) in Patients With Active ANCA-associated Vasculitis

The purpose of this study is to evaluate the efficacy and safety of iptacopan compared to standard of care (SOC) to induce and maintain remission in study participants with active granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA), when used in combination with rituximab (RTX) induction. The trial will also assess the impact of iptacopan on disease relapses, evolution of renal function and proteinuria, GC side effects, patients' immune status, and QoL.

Start Date05 Aug 2024

Sponsor / Collaborator

Novartis Pharmaceuticals Corp.

100 Clinical Results associated with Iptacopan

100 Translational Medicine associated with Iptacopan

100 Patents (Medical) associated with Iptacopan

Literatures (Medical) associated with Iptacopan

11 Dec 2024·EXPERT OPINION ON PHARMACOTHERAPY

Iptacopan for the treatment of paroxysmal nocturnal hemoglobinuria

Review

Author: Patel, Bhumika J. ; de Castro, Carlos M.

INTRODUCTION:

Standard-of-care first-line treatments for paroxysmal nocturnal hemoglobinuria (PNH) include the anti-C5 therapies eculizumab and ravulizumab. However, persistent anemia, likely due to extravascular hemolysis, and reduced quality of life (QoL) due to frequent infusions remain concerns. Iptacopan is a first-in-class oral proximal complement inhibitor that targets factor B in the alternative pathway (upstream of C5), limiting intravascular and extravascular hemolysis.

AREAS COVERED:

In patients previously treated with anti-C5 therapies or naive to complement inhibitors, iptacopan 200 mg twice daily resulted in clinically meaningful results in the pivotal phase 3 APPLY-PNH (NCT04558918) and APPOINT-PNH (NCT04820530) trials. Treatment with iptacopan was safe, and no treatment-related adverse events led to discontinuation.

EXPERT OPINION:

APPLY-PNH and APPOINT-PNH reported clinically meaningful improvements in hemoglobin, bilirubin, and lactate dehydrogenase levels; transfusion avoidance; reticulocyte count; and fatigue. Iptacopan's safety profile was comparable to other complement inhibitors. Oral iptacopan therapy allows patients to avoid infusions, limit clinical visits, decrease medical costs, improve anemia that persists with other complement inhibitors, and improve QoL. Long-term follow-up will further assess infections, thrombosis, and breakthrough hemolysis. Before treatment, physicians need to discuss current therapeutic options with patients for shared decision-making. Guidelines are being created to assist healthcare professionals in this advancing field.

01 Nov 2024·KAOHSIUNG JOURNAL OF MEDICAL SCIENCES

Complement factor B inhibitor LNP023 mediates the effect and mechanism of AMPK/mTOR on autophagy and oxidative stress in lupus nephritis

Article

Author: Zhang, Xi‐Mei ; Liu, Xin‐Kuo ; Peng, Liang ; Qing, Ming‐Jie

Abstract:

This study investigated the impact of LNP023 on the AMPK/mTOR signaling pathway in lupus nephritis (LN) and its effects on autophagy and oxidative stress. A mouse model of LN was established, and renal injury was confirmed by assessing various LN markers, including antinuclear antibody, ds‐DNA, anti‐Sm antibody, and others. Mice were treated with LNP023, the AMPK activator AICAR, or the AMPK inhibitor dorsomorphin. Renal injury and fibrosis were evaluated using HE and Masson staining. Expression levels of AMPK, mTOR, LC3, Beclin1, and p62 were assessed by immunohistochemistry and Western blot. Oxidative stress and inflammatory markers were measured by polymerase chain reaction and enzyme‐linked immunosorbent assay. LN mice exhibited low AMPK/p‐AMPK and high mTOR/p‐mTOR levels, alongside significant renal injury, fibrosis, reduced autophagy, and elevated oxidative stress. LNP023 treatment improved these parameters, with enhanced effects when combined with AICAR. Conversely, dorsomorphin reversed LNP023's therapeutic benefits. The complement factor B inhibitor LNP023 promotes kidney health in LN mice by mediating the AMPK/mTOR pathway, promoting autophagy, and attenuating oxidative stress.

01 Nov 2024·BIOMEDICAL CHROMATOGRAPHY

Pharmacokinetic study of iptacopan and its two acyl glucuronide metabolites in monkey plasma by liquid chromatography combined with electrospray ionization tandem mass spectrometry

Article

Author: Li, Jiacheng ; Li, Jingchu ; Liu, Shanshan ; Jia, Chenglin ; Ma, Chao ; Cao, Yongbin ; Zhang, Zhihui ; Chen, Jian

Abstract:

In this study, a simple and sensitive liquid chromatography tandem mass spectrometric method was developed and validated for the determination of iptacopan and two acyl glucuronidation metabolites in monkey plasma. The plasma sample was precipitated with acetonitrile and then separated on an Acquity UPLC BEH C18 column (2.1 × 100 mm, 1.7 μm) using 0.1% formic acid and 5 mM ammonium acetate in water and acetonitrile as the mobile phase. The mass spectrometry (MS) detection was performed in positive multiple reactions monitoring (MRM) mode with precursor‐to‐production transitions. The developed assay was validated over the range of 1–2000 ng/mL for three analytes with correlation coefficient (r) more than 0.99. The validation parameters including accuracy, precision, carryover effect, matrix effect, recovery, and stability were all within the acceptable limits. The validated method has been applied to investigate the pharmacokinetics of iptacopan and its two acyl glucuronidation metabolites in monkey plasma. After intravenous administration, iptacopan showed low clearance (2.75 mL/min/kg) in monkey plasma. After oral administration, the bioavailability was 55.43%. The exposure (AUC0−t) of direct acyl glucuronide (AG) of iptacopan accounts for 9.73% of the iptacopan plasma exposure. The AUC0−t of AG of dealkylated metabolite of iptacopan was present at a lower level, accounting for 0.5% of the iptacopan plasma exposure.

147

News (Medical) associated with Iptacopan

06 Dec 2024

·www.prnewswire.com

The Max Foundation (Max) Expands Collaboration with Novartis to Include Access to Innovative Treatment for Paroxysmal Nocturnal Hemoglobinuria (PNH)

Max to immediately begin process to provide humanitarian access for Fabhalta® (iptacopan) in collaboration with its network of partner hematologists across 53 countries People living with the rare blood disease in low-middle income countries require frequent blood transfusions, experience debilitating symptoms of fatigue and without treatment may progress to more serious life-threatening diseases The Max Foundation and Novartis have collaborated to improve access to medicines in low-resource countries for more than two decades SEATTLE, Dec. 6, 2024 /PRNewswire/ -- The Max Foundation (Max), a global nonprofit organization dedicated to accelerating health equity by delivering medication, technology, and supportive services to patients worldwide, today announced it will expand its collaboration with Novartis to include Fabhalta® (iptacopan), providing access to innovative treatment in low resource countries for PNH, a rare and debilitating blood disease that causes red blood cells to break apart. "We are thrilled to expand our reach to include people living with PNH in low-resource countries without access to an innovative treatment for this life-threatening condition," said Pat Garcia-Gonzalez, CEO of Max. "This collaboration reaffirms the deeply shared value of health equity for both organizations by making the latest treatment approved for the disease available at no cost, fulfilling our mission to accelerate health equity and enabling people to live with dignity and hope." PNH is caused by a genetic flaw that affects how red blood cells and platelets work. Only about 6 per 1 million people are diagnosed with it each year. People with PNH have sudden symptoms, including fatigue, weakness, increased heart rate, or shortness of breath, triggered by stressors like physical exertion or infection. During these episodes, red blood cells are broken down earlier than they should be, which causes patients to pass dark-colored urine. If PNH isn't treated, it can cause anemia, chronic kidney disease, or blood clots. "For more than 20 years, Novartis has successfully partnered with The Max Foundation to advance health equity by providing access to our innovative oncology medicines. Together, we have reached more than 100,000 patients by now, delivering sustainable and profound impact on communities around the globe. Novartis is proud to further expand our strong partnership, thereby accelerating early access for PNH patients in need," said Lutz Hegemann, President, Global Health at Novartis. The program will initially focus on identifying partner physicians ready to treat PNH and creating access pathways into 53 countries in Sub-Saharan Africa, Latin America, the Caribbean, and Southeast Asia and Asia Pacific. In addition to medication access, Max is seeking to collaborate with medical experts to strengthen health systems by providing education and training, identifying other potential collaborators in low- and middle-income countries, building capacity at medical institutions and facilitating conversations and partnership engagement to expand access to treatment. Max and Novartis have collaborated to improve access to innovative medicines since 2001. Starting with access to medicines for chronic myeloid leukemia and gastrointestinal stromal tumors, the collaboration has expanded to include advanced breast cancer and multiple rare diseases. Novartis is a member of Max's Humanitarian Partnership for Access to Critical Treatments (Humanitarian PACT), a collaboration among professional, nonprofit, and commercial organizations that share the commitment of the foundation to increase global access to treatment, care, and support for people living with cancer and other critical illnesses. Members of the Humanitarian PACT agree to invest resources and/or their unique organizational knowledge and capabilities to support the expansion of Max's proven treatment access model. About The Max Foundation The Max Foundation is a global health nonprofit organization dedicated to accelerating health equity. For 26 years, Max has pioneered practical, scalable, high-quality solutions to bring lifesaving treatments and patient-centered health care to more than 100,000 people living with cancer and critical illness in low- and middle-income countries. Max believes in a world where all people can access high-impact medicines, where geography is not destiny, and where everyone can strive for health with dignity and with hope. Learn more at . SOURCE The Max Foundation WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

06 Dec 2024

·endpts.com

Novartis bolsters Fabhalta data in competition with AstraZeneca

Novartis’ Fabhalta improved hemoglobin levels in patients with a rare blood disorder who switched from anti-C5 therapies, bolstering the drug’s case against AstraZeneca’s competing treatments. Fabhalta has been approved for exactly one year in paroxysmal nocturnal hemoglobinuria, a disorder that causes red blood cells to break down and can lead to anemia or blood clots. Some patients require transfusions. AstraZeneca’s C5 inhibitors Soliris and Ultomiris have long been the standard of care, each generating roughly $3 billion in 2023 sales. But on Friday, Novartis said patients in the single-arm Phase 3b APPULSE-PNH study saw their average hemoglobin levels improve from baseline over 24 weeks after switching from Soliris or Ultomiris, meeting the primary endpoint. “These data reinforce our confidence in Fabhalta, the first and only oral monotherapy currently available for the treatment of adults with PNH, to provide meaningful hemoglobin improvement, regardless of previous treatment experience,” David Soergel, global head of Novartis’ cardiovascular, renal and metabolism development unit, said in a news release . The company declined to provide specifics, though a spokesperson told Endpoints News that the data will be presented at a medical meeting in 2025. The results build on data from Novartis’ APPLY-PNH study. At last year’s American Society of Hematology conference, Novartis said that 94% of patients who switched from C5-inhibitors to Fabhalta in the study’s extension period achieved transfusion avoidance, and “mean hemoglobin levels increased to near-normal.” In that group, Novartis said mean hemoglobin levels were 9.1 g/dL at the start of the extension period, which is lower than patients’ average levels at the start of the APPULSE-PNH study. Soliris and Ultomiris are infusions, while Fabhalta is approved as a twice-daily oral pill. Novartis CEO Vas Narasimhan said in January that the company saw “very positive early launch signals” for Fabhalta, but added that he expected a “modest ramp for this brand given the dynamics within the PNH market,” according to an AlphaSense transcript. Fabhalta generated $44 million in third-quarter 2024 sales. In August, it was granted an accelerated approval in adults with primary immunoglobulin A nephropathy (IgAN), a rare autoimmune disease that affects the kidneys.

Clinical ResultDrug ApprovalPhase 3ASHImmunotherapy

06 Dec 2024

·www.globenewswire.com

New Phase IIIB data shows Novartis Fabhalta® improved hemoglobin levels in adult patients with paroxysmal nocturnal hemoglobinuria who switched from anti-C5 therapy

In the Phase IIIB APPULSE-PNH study, oral Fabhalta® (iptacopan) improved the average hemoglobin (Hb) level versus baseline in adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who were switched from anti-C5 therapies (Hb ≥10g/dL following treatment with eculizumab or ravulizumab)1,2PNH is a rare, chronic and serious complement-mediated blood disorder, characterized by hemolysis, anemia, thrombosis and other symptoms, and patients are often treated with anti-C5 therapies3-5Findings from APPULSE-PNH build on the Phase III program showing the efficacy and safety of Fabhalta in adults with PNH1, which is approved in the US, EU, Japan and China6-9 Basel, December 6, 2024 – Novartis today announced positive topline results from APPULSE-PNH, a Phase IIIB study evaluating the efficacy and safety of twice-daily oral monotherapy Fabhalta® (iptacopan) in adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who were switched from anti-C5 therapies (Hb ≥10g/dL following treatment with eculizumab or ravulizumab)1. After 24 weeks of treatment with Fabhalta, the average Hb level improved versus baseline1. “These new results add to the body of evidence reinforcing that Fabhalta can benefit both patients previously treated with anti-C5 therapies studied in the APPULSE-PNH and APPLY-PNH trials and complement-inhibitor naïve patients studied in the APPOINT-PNH trial,” said Antonio Risitano, M.D., Ph.D., Chair of the International PNH Interest Group and Head of the Hematology and Hematopoietic Transplant Unit, Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria at the AORN San Giuseppe Moscati, Avellino, Italy, and APPULSE-PNH trial lead investigator. “Treatment goals for patients with PNH have greatly evolved, and we can now aim to resolve signs and symptoms of disease in most patients. It is promising to see this evolution, and we will continue to make progress to best support these patients.” In the study, the safety profile of Fabhalta monotherapy was consistent with previously reported data1,10. “Across multiple clinical trials, Fabhalta has consistently shown clinically meaningful benefits for patients with PNH, and the APPULSE-PNH trial is a compelling addition to this body of evidence," said David Soergel, M.D., Global Head, Cardiovascular, Renal and Metabolism Development Unit, Novartis. “These data reinforce our confidence in Fabhalta, the first and only oral monotherapy currently available for the treatment of adults with PNH, to provide meaningful hemoglobin improvement, regardless of previous treatment experience.” Data will be presented at an upcoming medical meeting in 2025. Fabhalta was recently granted accelerated approval by the US Food and Drug Administration (FDA) for the reduction of proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, and development is ongoing in multiple complement-mediated diseases6,11. About APPULSE-PNH APPULSE-PNH (NCT05630001) is a Phase IIIB multicenter, single-arm, open-label study to evaluate the efficacy and safety of twice-daily oral Fabhalta® (iptacopan) monotherapy (200mg) in adults with PNH who were switched from anti-C5 therapies (eculizumab or ravulizumab)2. The trial enrolled 52 participants who received Fabhalta for 24 weeks2. Participants enrolled were required to be on a stable regimen with anti-C5 therapies (eculizumab or ravulizumab) for at least 6 months prior to screening with average hemoglobin (Hb) ≥10g/dL and no red blood cell transfusions in this period2,12. The primary endpoint is change from baseline Hb levels after 24 weeks of treatment with Fabhalta2,12. About paroxysmal nocturnal hemoglobinuria (PNH)PNH is a rare, chronic and serious complement-mediated blood disorder13. People with PNH have an acquired mutation in some of their hematopoietic stem cells (which are located in the bone marrow and can grow and develop into red blood cells [RBCs], white blood cells and platelets) that causes them to produce RBCs that are susceptible to premature destruction by the complement system4,13. This leads to intravascular hemolysis (destruction of RBCs within blood vessels) and extravascular hemolysis (destruction of RBCs mostly in the spleen and liver), which cause anemia (low levels of circulating RBCs), thrombosis (formation of blood clots), fatigue and other debilitating symptoms4,13.   It is estimated that approximately 10-20 people per million worldwide live with PNH13. Although PNH can develop at any age, it is often diagnosed in people between 30-40 years old14,15. PNH has a significant unmet need not addressed by anti-C5 therapies (eculizumab or ravulizumab). Anti-C5 therapies (eculizumab or ravulizumab) are commonly administered every 2-8 weeks as intravenous infusions, and treatment visits (including journey, waiting, infusion and recovery time) can take approximately 4 to 5 hours5. Despite treatment with anti-C5 therapies, a large proportion of people with PNH remain anemic, and some dependent on blood transfusions3,4,16-20. About Fabhalta® (iptacopan)  Fabhalta (iptacopan) is an oral, Factor B inhibitor of the alternative complement pathway12.  Fabhalta, discovered at Novartis, received approval by the FDA in December 2023 for the treatment of adults with PNH and by the European Medicines Agency (EMA) in May 2024 for the treatment of adults with PNH with hemolytic anemia6,7. In August 2024, Fabhalta was granted accelerated approval by the US Food and Drug Administration (FDA) for the reduction of proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, and development is ongoing in multiple complement-mediated diseases6,11. Fabhalta is being studied in a broad range of rare kidney diseases, including C3 glomerulopathy (C3G), atypical hemolytic uremic syndrome (aHUS), immune complex membranoproliferative glomerulonephritis (IC-MPGN) and lupus nephritis (LN). Studies are ongoing to evaluate the safety and efficacy profiles in these investigational indications and support potential regulatory submissions. DisclaimerThis media update 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 media update, 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 media update 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 media update as of this date and does not undertake any obligation to update any forward-looking statements contained in this media update 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 more than 250 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. References Novartis. Data on file.Clinicaltrials.gov. NCT05630001. Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan. (APPULSE). Available from: https://clinicaltrials.gov/study/NCT05630001 Accessed October, 2024.McKinley CE, Richards SJ, Munir T, et al. Extravascular Hemolysis Due to C3-Loading in Patients with PNH Treated with Eculizumab: Defining the Clinical Syndrome. Blood. 2017;130(Supplement 1):3471. Dingli D, Matos JE, Lehrhaupt K, et al. The burden of illness in patients with paroxysmal nocturnal hemoglobinuria receiving treatment with the C5-inhibitors eculizumab or ravulizumab: results from a US patient survey. Ann Hematol. 2022;101(2):251-263.Levy AR, Dysart L, Patel Y, et al. Comparison of Lost Productivity Due to Eculizumab and Ravulizumab Treatments for Paroxysmal Nocturnal Hemoglobinuria in France, Germany, Italy, Russia, Spain, the United Kingdom, and the United States. Blood. 2019;134(Supplement_1):4803.Fabhalta®. US FDA Prescribing information. East Hanover, NJ:Novartis Pharmaceuticals Corp; 2024. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/218276s001lbl.pdf Accessed November 2024.Fabhalta®. EMA Summary of Product Characteristics. Novartis Europharm Limited; 2024. Available from: https://www.ema.europa.eu/en/documents/product-information/fabhalta-epar-product-information_en.pdf Accessed November 2024.Fabhalta®. PMDA Drug Information Sheet. Novartis Pharma KK; 2024. Available from: https://www.pmda.go.jp/files/000271929.pdf Accessed November 2024.Fabhalta®. NMPA drug insert. Novartis Pharma Schweiz AG; 2024. Available from: https://www.ccfdie.org/en/gzdt/webinfo/2024/05/1716632195327630.html Accessed November 2024.Dighriri IM, Al-Qahtani RM, Almutairi AO, et al. Iptacopan Efficacy and Safety to Treat Paroxysmal Nocturnal Hemoglobinuria (PNH): A Systematic Review and Meta-Analysis. Cureus. 2024;16(8):e67830. Novartis. Press release. Novartis receives FDA accelerated approval for Fabhalta® (iptacopan), the first and only complement inhibitor for the reduction of proteinuria in primary IgA nephropathy (IgAN). Available from: 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, 2024.Risitano AM, Araten DJ, Kuter D, et al. Pb2063: APPULSE-PNH: A phase IIIB trial to evaluate the efficacy and safety of switching to iptacopan in patients with paroxysmal nocturnal hemoglobinuria (PNH) on anti-c5 therapy with hemoglobin >10g/dl. Hemasphere. 2023;7(Suppl):e08587b7.Cançado RD, Araújo ADS, Sandes AF, et al. Consensus statement for diagnosis and treatment of paroxysmal nocturnal haemoglobinuria. Hematol Transfus Cell Ther. 2021;43(3):341-348.Hill A, DeZern AE, Kinoshita T, Brodsky RA. Paroxysmal nocturnal haemoglobinuria. Nat Rev Dis Primers. 2017;3:17028.Röth A, Maciejewski J, Nishimura JI, Jain D, Weitz JI. Screening and diagnostic clinical algorithm for paroxysmal nocturnal hemoglobinuria: Expert consensus. Eur J Haematol. 2018;101(1):3-11.Risitano AM, Marotta S, Ricci P, et al. Anti-complement Treatment for Paroxysmal Nocturnal Hemoglobinuria: Time for Proximal Complement Inhibition? A Position Paper From the SAAWP of the EBMT. Front Immunol. 2019;10:1157.Shammo et al. HemaSphere 2023 Shammo J, Kim J, Georget M, et al. P796: Hospitalization in patients with paroxysmal nocturnal hemoglobinuria: a retrospective analysis of observational study data from the United States. Hemasphere. 2023;7(Suppl):e22585a2.Debureaux PE, Kulasekararaj AG, Cacace F, et al. Categorizing hematological response to eculizumab in paroxysmal nocturnal hemoglobinuria: a multicenter real-life study. Bone Marrow Transplant. 2021;56(10):2600-2602.Schrezenmeier H, Kulasekararaj A, Mitchell L, et al. One-year efficacy and safety of ravulizumab in adults with paroxysmal nocturnal hemoglobinuria naïve to complement inhibitor therapy: open-label extension of a randomized study. Ther Adv Hematol. 2020;11:2040620720966137.Young NS, Meyers G, Schrezenmeier H, Hillmen P, Hill A. The management of paroxysmal nocturnal hemoglobinuria: recent advances in diagnosis and treatment and new hope for patients. Semin Hematol. 2009;46(1 Suppl 1):S1-S16. # # # Novartis Media RelationsE-mail: media.relations@novartis.com Central North America Anja von Treskow +41 79 392 9697 Michael Meo +1 862 274 5414 Anna Schäfers +41 79 801 7267 SwitzerlandSatoshi Sugimoto +41 79 619 2035 Novartis Investor RelationsCentral investor relations line: +41 61 324 7944E-mail: investor.relations@novartis.com Central North America Isabella Zinck +41 61 324 7188 Sloan Simpson +1 862 345 4440 Nicole Zinsli-Somm +41 61 324 3809 Jonathan Graham +1 201 602 9921 Imke Kappes +41 61 324 8269 Parag Mahanti +1 973 876 4912

Phase 3Clinical ResultDrug ApprovalAccelerated ApprovalNDA

100 Deals associated with Iptacopan

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KEGG	Wiki	ATC	Drug Bank
-	Iptacopan	-	DB16200

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Glomerulonephritis, IGA	US	Novartis Pharmaceuticals Corp.	07 Aug 2024
Hemoglobinuria, Paroxysmal	US	Novartis Pharmaceuticals Corp.	05 Dec 2023

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
C3 glomerulopathy	NDA/BLA	CN	Novartis Pharma Schweiz AG	05 Sep 2024
C3 glomerulopathy	NDA/BLA	CN	Novartis Pharma Produktions GmbH	05 Sep 2024
Myasthenia Gravis	Phase 3	US	Novartis Pharma AG	31 Jul 2024
Myasthenia Gravis	Phase 3	US	Novartis Pharmaceuticals Corp.	31 Jul 2024
Myasthenia Gravis	Phase 3	JP	Novartis Pharmaceuticals Corp.	31 Jul 2024
Myasthenia Gravis	Phase 3	JP	Novartis Pharma AG	31 Jul 2024
Myasthenia Gravis	Phase 3	DK	Novartis Pharma AG	31 Jul 2024
Myasthenia Gravis	Phase 3	DE	Novartis Pharma AG	31 Jul 2024
Myasthenia Gravis	Phase 3	GR	Novartis Pharma AG	31 Jul 2024
Myasthenia Gravis	Phase 3	GR	Novartis Pharmaceuticals Corp.	31 Jul 2024

Clinical Result

Indication

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ASH2024	Not Applicable	Hemoglobinuria, Paroxysmal	-	Iptacopan monotherapy 200 mg twice daily	fbdzjhurqs(iyrbsltwhj) = djwkoylmuq qnikqzmjfd (hrkndegtuh, 87.4) View more	-	09 Dec 2024
				Eculizumab	fbdzjhurqs(iyrbsltwhj) = fbusyuudbi qnikqzmjfd (hrkndegtuh, 77.2) View more
ASH2024	Not Applicable	Cold Agglutinin Disease	-	Iptacopan 200 mg	epdjjmhbef(sdnoxtgrpz) = Eight pts (80%) experienced ≥1 treatment-emergent adverse event (TEAE), most of which were mild in severity. Two pts discontinued treatment because of TEAEs (1 had increased alanine aminotransferase and aspartate aminotransferase [suspected to be treatment related]; 1 had recurrent breast cancer [not suspected to be treatment related]). Two pts had serious adverse events (1 had increased blood creatinine and acute kidney injury; 1 had spinal fracture), but none were suspected to be treatment related. oskayzhjxl (dfbmwwogqc )	-	08 Dec 2024
NCT04578834 (APPLAUSE-IgAN, FDA_CDER) Manual	Phase 3	Glomerulonephritis, IGA	250	FABHALTA	zqmcjmdmtf(flbjdrnovp) = dmxvdxkiww bbxluxyhrs (ddzksvuuka, 36 - 51) View more	Positive	07 Aug 2024
				Placebo	zqmcjmdmtf(flbjdrnovp) = lpeslmsbwc bbxluxyhrs (ddzksvuuka, -5 to 21) View more
NCT04154787 (CTgov)	Phase 2	Idiopathic Membranous Glomerulonephritis	37	Rituximab	mzadfwldef(ogfltjmmnu) = rrmpgdrflv ykqqqfcdlb (vnsdgalxtg, mgbnjwmila - jotaenvfvd) View more	-	05 Jul 2024
NCT04817618 (APPEAR-C3G, PRNewswire 1 \| PRNewswire 2) Manual	Phase 3	C3 glomerulopathy	-	Fabhalta (iptacopan)	ixhzeollol(wbwdjnlzix) = ncbgeixkbw ieyqhvhcra (ruhckuuukb )	Positive	25 May 2024
				Placebo	-
NCT04820530 (APPOINT-PNH, EHA2024) Manual	Phase 3	Hemoglobinuria, Paroxysmal	40	Iptacopan 200 mg	fnitkggpli(egapvxdlpz) = yafwotnfry krcwphxjpi (ubxuizuweg ) View more	Positive	14 May 2024
NCT04558918 \| NCT03500549 (APPLY-PNH \| PEGASUS, EHA2024) Manual	Phase 3	Hemoglobinuria, Paroxysmal Maintenance	103	Iptacopan	nlzembqtne(mbeycnbiqw) = czwsmpysmy efsyvhtsbv (bpxpnmehsb ) View more	Positive	14 May 2024
				Pegcetacoplan	nlzembqtne(mbeycnbiqw) = xpvdqslkdb efsyvhtsbv (bpxpnmehsb )
NCT04747613 (APPOINT-PNH \| APPLY-PNH \| roll-over extension, EHA2024)	Phase 3	Hemoglobinuria, Paroxysmal	61	Iptacopan	pmemurppgg(pvuzemybdr) = fhmdagtajq matslqccrw (hznkxwmlzb )	Positive	14 May 2024
NCT04558918 (APPLY-PNH, EHA2024)	Phase 3	Hemoglobinuria, Paroxysmal C3 fragment deposition	95	Iptacopan 200 mg twice daily	luvzqqwhrp(lnzactgezz) = sfezdwhzff ltujpwblaq (cdowpshloo, -16.1%) View more	Positive	14 May 2024
				Anti-C5 inhibitors	luvzqqwhrp(lnzactgezz) = kfyyvedlzh ltujpwblaq (cdowpshloo, -16.9%) View more
NCT04578834 (APPLAUSE-IgAN, PRNewswire) Manual	Phase 3	Glomerulonephritis, IGA	443	Fabhalta	idldrzddae(ertktlujpn) = xbwqrwsrnd vkieiomrch (cnlaexkgsy )	Positive	15 Apr 2024

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