RegulationBreakthrough Therapy (United States), Accelerated Approval (United States), Orphan Drug (United States), Rare Pediatric Disease (United States), Orphan Drug (European Union), Breakthrough Therapy (China), Orphan Drug (Japan), Orphan Drug (South Korea), Orphan Drug (Australia), Priority Review (Australia), Priority Review (United States), PRIME (European Union), Conditional marketing approval (China), Priority Review (China)

Structure/Sequence

Molecular FormulaC25H33ClN2O5

InChIKeyJUWBBUFSAGEROP-VVJLZRNGSA-N

CAS Registry2447007-60-3

Clinical Trials associated with Iptacopan

NCT06994845

/ RecruitingPhase 3

A Single-arm, Multicenter, Phase III Study to Assess Efficacy, Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric Patients of 2 to <18 Years of Age With Primary Immunoglobulin A Nephropathy (IgAN)

The study is an open-label, single arm, multicenter, Phase III study to determine proteinuria reduction, pharmacokinetics (PK), safety and tolerability (including CV surveillance) of iptacopan in primary immunoglobulin A nephropathy (IgAN) pediatric patients aged 2 to <18 years.

Start Date27 Nov 2025

Sponsor / Collaborator

Novartis Pharmaceuticals Canada, Inc.

NCT06934967

/ RecruitingPhase 3

An Open-label, Single-arm, Multicenter, Phase 3 Study to Assess Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric PNH Patients 2 to <18 Years of Age

The purpose of this open-label, single arm, multicenter, phase 3 study is to assess the pharmacokinetics of iptacopan in pediatric patients and to assess whether iptacopan is safe and well tolerated when used for the treatment of pediatric paroxysmal nocturnal hemoglobinuria (PNH) patients 2 to < 18 years of age.

Start Date28 Oct 2025

Sponsor / Collaborator

Novartis Pharmaceuticals Canada, Inc.

NCT06931691

/ RecruitingNot Applicable

A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan on Disease Management, Treatment-Related Outcomes and Healthcare Resource Utilization for Adult Patients With Paroxysmal Nocturnal Hemoglobinuria in China

The implementation of new standards for the management of PNH and the use of iptacopan in patients with PNH are expected to change the treatment landscape and improve the overall prognosis of patients.
Based on these backgrounds, we plan to conduct a real-world study of iptacopan to further evaluate its impact on treatment-related outcomes, disease management, and healthcare resource utilization in Chinese patients with PNH.

Start Date10 Jun 2025

Sponsor / Collaborator

Novartis Pharmaceuticals Canada, Inc.

100 Clinical Results associated with Iptacopan

100 Translational Medicine associated with Iptacopan

100 Patents (Medical) associated with Iptacopan

105

Literatures (Medical) associated with Iptacopan

31 Dec 2025·Hematology

Severe hemolysis flare of refractory autoimmune hemolytic anemia with positive complement component C3d responsive to Iptacopan with cyclophosphamide and prednisone: a case report

Article

Author: He, Guangsheng ; Zhang, Yawen ; Gong, Yuemin

OBJECTIVES:

Autoimmune hemolytic anemia (AIHA) is characterized by autoimmune-mediated destruction of erythrocytes. Both AIHA and Evans syndrome are rare, manifesting a severe clinical course, high relapse rate, and potentially fatal outcomes. Refractory AIHA shows poor responsiveness to multiple treatment regimens, and there is still a lack of effective treatment regimens for serious hemolytic episodes.

METHODS:

Three refractory AIHA cases with a positive complement component C3d and hemolytic flare were treated with the oral factor B inhibitor ptacopan in conjunction with cyclophosphamide and prednisone.

RESULTS:

After treatment with iptacopan plus cyclophosphamide and prednisone, all three patients showed a rapid increase in Hb levels, a decrease in reticulocyte proportion, and a significant reduction in hemolysis manifestations (Lower LDH level and unconjugated bilirubin).

CONCLUSION:

Three refractory cases of AIHA showed good therapeutic efficacy after treatment with iptacopan combined with cyclophosphamide and prednisone. These cases provide a potentially effective treatment option for severe hemolytic episodes in refractory AIHA.

31 Dec 2025·Hematology

Successful switch from pegcetacoplan to iptacopan after repeated severe breakthrough hemolysis events – case report

Article

Author: Füreder, Wolfgang ; Reinisch, Andreas

OBJECTIVES:

A subset of paroxysmal nocturnal hemoglobinuria (PNH) patients develops clinically relevant extravascular hemolysis when treated with complement C5 inhibitors. These patients may benefit proximal complement inhibitors such as pegcetacoplan, danicopan or iptacopan. No studies comparing these substances are available. Breakthrough hemolysis (BTH) - defined by exacerbation of intravascular hemolysis despite complement inhibition - can be severe especially in patients treated with proximal complement inhibitors.

METHODS:

We report on a PNH patient who had suffered repeated episodes of severe BTH with acute renal failure during 1 year of treatment with pegcetacoplan. The patient was switched to iptacopan and followed also for 1 year.

RESULTS:

After switching to iptacopan, no further BTH occurred for the duration of 12 months follow up. The patient maintained stable hemoglobin and reticulocyte counts as well as lactate dehydrogenase (LDH) levels within the normal range.

DISCUSSION:

Despite dose escalation of pegcetacoplan, BTH recurred in our patient. Therefore, an alternative therapy was warranted. During iptacopan therapy - chosen due to patient preference -no further BTH occurred. However, more data from a larger number of patients are needed.

CONCLUSION:

A switch to iptacopan may be an option for pegcetacoplan treated patients who experience repeated BTH in spite of dose escalation.

01 Nov 2025·DRUGS

Pharmacological Therapies in Paroxysmal Nocturnal Haemoglobinuria: Focus on Complement Inhibition

Review

Author: Holt, Matthew ; Szer, Jeff ; Kelly, Richard J

Paroxysmal nocturnal haemoglobinuria (PNH) is an ultra-rare acquired genetic stem cell disorder based on a mutation in the PIGA gene that results in susceptibility of resulting blood cells to complement-mediated intravascular haemolysis (IVH). In countries where anti-complement therapy is available, pharmacological treatments have transformed this disease from a highly morbid and sometimes lethal disorder. The first treatment developed was the terminal complement (C5) monoclonal antibody inhibitor eculizumab, in 2002. This has been largely supplanted by a longer-acting antibody, ravulizumab, targeting the same binding site on C5. These agents significantly modify the natural history of the disease by reducing the risk of thrombosis, the most lethal complication of PNH, as well as reducing transfusion dependence and improving renal function, quality of life and probably, survival. Other terminal inhibitors available include eculizumab biosimilars, crovalimab, pozelimab and cemdisiran (combination). Despite this, a proportion of patients develop extravascular haemolysis (EVH) based on the accumulation of C3 components on these PNH blood cells, which no longer undergo IVH because of C5 inhibition. This has led to the development of proximal complement inhibitors, which have been generally successful at reducing this iatrogenic complication, improving haemoglobin concentrations, reducing transfusion dependency and improving quality of life. Currently available proximal inhibitors (and their targets) are pegcetacoplan (C3), danicopan (Factor D) and iptacopan (Factor B). While effective, as with all other complement inhibitors, there is a risk of breakthrough IVH with their use and approaches to manage this complication are being developed.

242

News (Medical) associated with Iptacopan

25 Nov 2025

·endpoints.news

Otsuka’s anti-APRIL antibody gets FDA approval for kidney disease IgAN

Otsuka secured FDA accelerated approval for its antibody drug in a disease that impacts the kidneys’ ability to filter waste. The Japanese drugmaker said late Tuesday the agency greenlit sibeprenlimab for IgA nephropathy (IgAN). The decision was based on data from a Phase 3 trial called VISIONARY in which sibeprenlimab reduced levels of protein in the urine, also known as proteinuria, by just over half versus placebo. Proteinuria is a biomarker of kidney function. Otsuka obtained the rights to sibeprenlimab during its $430 million acquisition of Visterra in 2018. The drug will be marketed as Voyxact. Pricing information wasn’t immediately available. Voyxact is the first drug of its class to clinch an approval in IgAN. It works by blocking a ligand called APRIL which is believed to drive the production of the abnormal IgA protein that builds up in the kidneys. These protein deposits damage the delicate filters that run through the kidneys, causing tissue damage and scarring. “We’re really hoping that in addition to improving symptoms, [Voyxact] will actually improve long-term kidney outcomes and has the potential for disease modification,” Otsuka chief medical officer John Kraus told Endpoints News in an interview prior to the approval. Vera Therapeutics is also advancing an APRIL-directed drug for IgAN called atacicept. The biotech has shared Phase 3 data and filed its drug for accelerated approval. Atacicept also targets a different family of ligands called BAFF. But Voyxact is arguably the more convenient option thanks to its once-monthly injection schedule, compared with atacicept’s once-weekly regimen. Meanwhile, Vertex is also advancing a dual APRIL/BAFF inhibitor called povetacicept that it says has “pipeline-in-a-product” potential. The IgAN market is already highly competitive. Novartis’ factor B inhibitor Fabhalta won accelerated approval for IgAN in August 2024, and cleared a confirmatory trial last month. In April, the Swiss company’s endothelin A receptor antagonist Vanrafia also clinched an accelerated approval for IgAN. Travere Therapeutics’ Filspari and Calliditas Therapeutics’ Tarpeyo have been on the market for some years. All four of the drugs are taken orally, which patients generally tend to prefer over injections. How well Voyxact does commercially could also depend on estimated glomerular filtration rate data from the VISIONARY study, which is expected in early 2026. This is a more robust measure of kidney function compared with proteinuria. The company says that data is intended to support traditional approval.

Drug ApprovalPhase 3Accelerated ApprovalClinical ResultAcquisition

25 Nov 2025

·www.globenewswire.com

Novartis data underscore pioneering scientific innovation in Hematology and Oncology at ASH and SABCS

Positive results from ianalumab pivotal Phase III trial in ITP patients previously treated with corticosteroids to be presented as late-breakerScemblix data across clinical and real-world settings offer new evidence informing CML care amid evolving patient needs 96-week pelabresib Phase III data represent longest follow-up of first-line myelofibrosis patients in randomized combination trialKisqali NATALEE and MONALEESA data add to evidence of long-term benefits for early and metastatic breast cancer patients Basel, November 25, 2025 – Novartis will present data from over 70 abstracts, including investigator-initiated trials at the 67th American Society of Hematology (ASH) Annual Meeting & Exposition and 2025 San Antonio Breast Cancer Symposium® (SABCS). Featured among these latest advances in hematology and oncology are 11 oral presentations, with the Phase III VAYHIT2 trial for ianalumab in immune thrombocytopenia (ITP) accepted as a late-breaker abstract.“For decades, Novartis has redefined the future of hematology and oncology, and we’re building on that foundation with compelling new data presented at ASH and SABCS,” said Mark Rutstein, M.D., Global Head, Oncology Development, Novartis. “These data underscore how we seek to set new standards for transformative care, with the aim of turning cutting-edge innovation into meaningful impact for patients.”Key highlights of data accepted by ASH include: Abstract Title Abstract Number/ Presentation Details Ianalumab (VAY736)Primary results from VAYHIT2, a randomized, double-blind, Phase 3 trial of ianalumab plus eltrombopag versus placebo plus eltrombopag in patients with primary immune thrombocytopenia (ITP) who failed first-line corticosteroid treatmentAbstract #LBA-2Oral PresentationDecember 9, 7:45 – 8:00 am ETSecondary analysis results from VAYHIT3, a Phase 2 study of ianalumab in patients with primary immune thrombocytopenia previously treated with at least two lines of therapyAbstract #844Oral PresentationDecember 8, 3:30 – 3:45 pm ETScemblix® (asciminib)Asciminib (ASC) demonstrates continued improvement in patient-reported outcomes (PROs) vs investigator-selected tyrosine kinase inhibitors (IS-TKIs) in newly diagnosed chronic myeloid leukemia (CML): ASC4FIRST week 96 analysisAbstract #1997Poster PresentationDecember 6, 5:30 – 7:30 pm ETImproved long-term tolerability with asciminib (ASC) vs investigator-selected (IS) tyrosine kinase inhibitors (TKIs) in patients (pts) with newly diagnosed chronic myeloid leukemia in chronic phase (CML-CP): Week 96 exploratory analysis of the phase 3 ASC4FIRST trialAbstract #5549Poster PresentationDecember 8, 6:00 – 8:00 pm ETAsciminib (ASC) in chronic myeloid leukemia in chronic Phase (CML-CP): Efficacy and safety results of the Phase 2 ASC2ESCALATE trial in the cohort of patients (pts) with 1 prior tyrosine kinase inhibitor (TKI)Abstract #906Oral PresentationDecember 8, 4:00 – 4:15 pm ETA comparison of real-world outcomes of asciminib versus ATP-competitive tyrosine kinase inhibitors as second-line treatment in patients with chronic myeloid leukemia in chronic phaseAbstract #724Oral PresentationDecember 7, 5:15 – 5:30 pm ETPelabresib (DAK539)Durable efficacy and long-term safety with pelabresib plus ruxolitinib in JAK Inhibitor–Naive myelofibrosis: 96-week Results from the Phase III MANIFEST-2 studyAbstract #910Oral PresentationDecember 8, 3:30 – 3:45pm ETRapcabtagene autoleucel (YTB323)Rapcabtagene autoleucel (YTB323) for patients with first line high-risk large B-cell lymphoma: phase II interim resultsAbstract #670Oral PresentationDecember 7, 5:15 – 5:30 pm ETFabhalta® (iptacopan)Oral iptacopan monotherapy demonstrates clinically meaningful hemoglobin increases in patients with paroxysmal nocturnal hemoglobinuria with baseline hemoglobin levels 10 to <12 g/dL on anti-C5 therapy: Subgroup analysis of the APPULSE-PNH Phase 3b trialAbstract #4981Poster PresentationDecember 8, 6:00 – 8:00 pm ETLong-term safety and efficacy of iptacopan in patients with paroxysmal nocturnal hemoglobinuria: 4- and 5-year follow-up of patients from phase 2 studies who entered the roll-over extension programAbstract #3198Poster PresentationDecember 7, 6:00 – 8:00 pm ETThe 2-year efficacy and safety of iptacopan monotherapy in patients with paroxysmal nocturnal hemoglobinuria with a history of aplastic anemia on concomitant immunosuppressive therapy who entered the roll-over extension programAbstract #4978Poster PresentationDecember 8, 6:00 – 8:00 pm ET Key highlights of data accepted by SABCS include: Kisqali® (ribociclib)Pooled analysis of patients (pts) treated with 1st-line (1L) ribociclib (RIB) + endocrine therapy (ET) in the MONALEESA (ML) studies: long-term progression-free survival (PFS)Abstract # PD5-10Poster Spotlight PresentationDecember 11, 8:09 – 8:12 am CSTFive-year analysis of distant disease-free survival (DDFS) across key subgroups from the phase 3 NATALEE trial of ribociclib (RIB) plus a nonsteroidal aromatase inhibitor (NSAI) in patients with HR+/HER2− early breast cancer (EBC)Abstract # PS3-09-08Poster PresentationDecember 11, 12:30 – 2:00 pm CSTProgression-free survival (PFS) and overall survival (OS) results from the phase 3 MONALEESA-3 trial of postmenopausal patients with hormone receptor–positive (HR+)/HER2-negative (HER2−) advanced breast cancer (ABC) treated with ribociclib (RIB) + fulvestrant (FUL): A subgroup analysis of patients with invasive lobular carcinoma (ILC) Abstract # PS1-10-27 Poster PresentationDecember 10, 12:30 – 2:00 pm CSTRibociclib drug-drug interaction and concomitant medication management in early and advanced breast cancer patientsAbstract # PS3-09-15Poster PresentationDecember 11, 12:30 – 2:00 pm CSTReal-world patient (pt) and caregiver experiences with breast cancer (BC) risk of recurrence (ROR) in the US: Results of an Online Survey and Social Media Analysis Abstract # PS1-04-17Poster PresentationDecember 10, 12:30 – 2:00 pm CSTRepower: a real-world noninterventional study of outcomes and experiences in patients with hormone receptor-positive (HR+)/human epidermal growth fact receptor 2-negative (HER2−) early breast cancer (EBC) treated with an adjuvant cyclin-dependent kinase 4 and 6 inhibitor (CDK4/6i) plus endocrine therapy (ET) Abstract # PS3-08-27Poster PresentationDecember 11, 12:30 – 2:00 pm CST 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 hematologyOur legacy in hematology runs deep, shaped by over 25 years of progress, partnerships and a commitment to keep asking questions, challenging norms and striving for better answers in a uniquely complex field. In the past two decades, we have delivered more than 10 medicines across more than 15 blood cancers and serious blood disorders including leading the era of targeted therapies in cancer and bringing the first CAR-T therapy to patients. Innovation in hematology has brought significant progress, yet patients and clinicians continue to face persistent challenges. We’re forging the future of hematology, powered by our foundation in scientific discovery to deliver meaningful change for patients with unmet needs.Novartis in breast cancer For over 30 years, Novartis has been at the forefront of driving scientific advancements for individuals affected by breast cancer and enhancing clinical practice in collaboration with the global community. With one of the most comprehensive breast cancer portfolios and pipeline, Novartis leads the industry in discovery of new therapies and combinations in HR+/HER2- breast cancer, the most common form of the disease. 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 Media RelationsE-mail: media.relations@novartis.comNovartis Investor RelationsCentral investor relations line: +41 61 324 7944E-mail: investor.relations@novartis.com

Phase 2Phase 3Clinical ResultASHImmunotherapy

20 Nov 2025

·firstwordpharma.com

Novartis ups peak sales view for Kisqali, Scemblix

Novartis on Thursday upped peak sales estimates for two of its key cancer drugs, Kisaqali and Scemblix, which together are now forecast to bring in revenue of at least $14 billion. The upgrade for the two products came alongside the company disclosing new mid-term guidance, which sees annual sales growth in the range of 5% to 6% on a constant currency basis between now and 2030 — previously, growth of 6% annually was expected from 2024 to 2029. CEO Vas Narasimhan said "we expect to sustain…momentum over the next five years, driven by assets we already have in hand as well as upcoming launches with multi-billion-dollar sales potential." According to the drugmaker, it now has eight derisked assets on the market that each have peak sales potential of between $3 billion and $10 billion — Kisqali, Cosentyx, Kesimpta, Pluvicto, Scemblix, Leqvio, Fabhalta and Rhapsido. These are forecast to more than offset the impact from generic erosion through 2030, from competition to products such as Cosentyx, Entresto and Tasigna.For Kisqali, peak sales are expected to be at least $10 billion, up from an earlier estimate of $8 billion, while revenue from Scemblix is seen reaching at least $4 billion, lifted from $3 billion previously.Beyond its launched medicines, Novartis said Thursday that it expects at least 15 potentially submission-enabling clinical readouts over the next two years. These include: ianalumab; Ac-PSMA-617; OAV101 IT; pelacarsen; del-desiran and del-brax.

Drug ApprovalBiosimilar

100 Deals associated with Iptacopan

External Link

KEGG	Wiki	ATC	Drug Bank
-	Iptacopan	-	DB16200

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
C3 glomerulopathy	United States	Novartis Pharmaceuticals Corp.	20 Mar 2025
Glomerulonephritis, IGA	United States	Novartis Pharmaceuticals Corp.	07 Aug 2024
Hemoglobinuria, Paroxysmal	United States	Novartis Pharmaceuticals Corp.	05 Dec 2023

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Complement Factor H Deficiency	NDA/BLA	European Union	Novartis Europharm Ltd.	27 Feb 2025
Myasthenia Gravis	Phase 3	United States	Novartis Pharma AG	31 Jul 2024
Myasthenia Gravis	Phase 3	United States	Novartis Pharmaceuticals Canada, Inc.	31 Jul 2024
Myasthenia Gravis	Phase 3	China	Novartis Pharmaceuticals Canada, Inc.	31 Jul 2024
Myasthenia Gravis	Phase 3	Japan	Novartis Pharma AG	31 Jul 2024
Myasthenia Gravis	Phase 3	Japan	Novartis Pharmaceuticals Canada, Inc.	31 Jul 2024
Myasthenia Gravis	Phase 3	Denmark	Novartis Pharma AG	31 Jul 2024
Myasthenia Gravis	Phase 3	Denmark	Novartis Pharmaceuticals Canada, Inc.	31 Jul 2024
Myasthenia Gravis	Phase 3	Germany	Novartis Pharma AG	31 Jul 2024
Myasthenia Gravis	Phase 3	Greece	Novartis Pharmaceuticals Canada, Inc.	31 Jul 2024

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05630001 (APPULSE \| APPULSE-PNH, CTgov)	Phase 3	Hemoglobinuria, Paroxysmal	52	Iptacopan	nrnpfhaafx(wshlflnwbq) = awfdsuqexv fezjhudsoh (lzgwoafqvk, nzhjfabsww - ghbezsdaeg) View more	-	15 Dec 2025
NCT04747613 (PNH-REP, ASH2025)	Phase 3	Hemoglobinuria, Paroxysmal	26	Iptacopan	ujjxswbzqr(wpflhdjoze) = tircmknjcj fehxwkfcku (wmouxyqsuz ) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Hemoglobinuria, Paroxysmal	18	Iptacopan monotherapy	xizmvdfbep(yympzhamev) = yherypudqi gyodlddigh (vaomjlmspa, 10.02 - 55.98) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Hemoglobinuria, Paroxysmal	31	Iptacopan	wcgbxmadxy(gfjcnopyor) = zowzuoqkxw zspbclbael (hjahihkzcl ) View more	Positive	06 Dec 2025
				Eculizumab	wcgbxmadxy(gfjcnopyor) = exgyouibli zspbclbael (hjahihkzcl ) View more
ASH2025	Not Applicable	Hemoglobinuria, Paroxysmal	62	Iptacopan (Treatment-naive patients)	bpbwqpyomk(laqybaxcdg) = Four patients discontinued iptacopan (2 pregnancies, 1 insurance reversal, 1 intolerable gastrointestinal toxicity). Three patients reported headaches, and 1 reported abdominal pain; all resolved within 1 week from treatment start. lzibsfopyk (evpmzkvktk ) View more	Positive	06 Dec 2025
				Iptacopan (Switched from prior therapy)
ASH2025	Not Applicable	Hemoglobinuria, Paroxysmal	15	Pegcetacoplan	tkkecfifde(wttxdvdpjv) = nnyavhusmn nyzjnuwbkn (wziqcfovpq, 1.8) View more	Positive	06 Dec 2025
				Iptacopan	tkkecfifde(wttxdvdpjv) = ukqraqbvzs nyzjnuwbkn (wziqcfovpq, 2.1) View more
ASH2025	Not Applicable	Hemoglobinuria, Paroxysmal	20	Iptacopan monotherapy (200 mg bid)	urywvxnixt(cfksgnuffl) = ejdfvlatsw pewkvmgden (jnbaeiexym ) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Hemoglobinuria, Paroxysmal	42	Iptacopan	vetrnxcoix(hndgogpppz) = ailcsuyarz dusjopubro (ufszzlfvmd ) View more	Positive	06 Dec 2025
				Iptacopan (complement inhibitor-naive patients)	pdwipqdrmb(ydyibvludv) = msbcrghhgw ecrucxwxyc (hmdtywqzyo ) View more
NCT03955445 (APPEAR-C3G, ASN2025) Manual	Phase 3	C3 glomerulopathy	66	Iptacopan	jznzrgfilx(zuohwcuqbx) = ussijnefro cawurheafu (tyrtdlosnh ) View more	Positive	07 Nov 2025
				placebo-iptacopan switch	jznzrgfilx(zuohwcuqbx) = wesfhgaomy cawurheafu (tyrtdlosnh ) View more
NCT04578834 (APPLAUSE-IgAN, GlobeNewswire) Manual	Phase 3	Glomerulonephritis, IGA	477	Fabhalta	qnwemfuxcy(aoermatygr) = Fabhalta demonstrated statistically significant, clinically meaningful superiority compared to placebo in slowing IgAN progression measured by annualized total slope of eGFR decline over two years. laeamzswmr (bqdwsdseex ) Met View more	Positive	16 Oct 2025
				Placebo

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