RegulationPriority Review (United States), Breakthrough Therapy (United States), Accelerated Approval (United States), Orphan Drug (United States), Orphan Drug (European Union), PRIME (European Union), Breakthrough Therapy (China), Orphan Drug (South Korea), Orphan Drug (Australia), Priority Review (Australia), Rare Pediatric Disease (United States), Priority Review (China)

Structure/Sequence

Molecular FormulaC25H33ClN2O5

InChIKeyJUWBBUFSAGEROP-VVJLZRNGSA-N

CAS Registry2447007-60-3

Clinical Trials associated with Iptacopan

NCT06994845

/ Not yet 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 Date08 Dec 2025

Sponsor / Collaborator

Novartis Pharmaceuticals Canada, Inc.

NCT06934967

/ Not yet 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 Jul 2025

Sponsor / Collaborator

Novartis Pharmaceuticals Canada, Inc.

NCT06931691

/ Not yet 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 Date30 Apr 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

Literatures (Medical) associated with Iptacopan

03 Jun 2025·PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Host complement C3 promotes malaria transmission by killing symbiotic bacteria in the mosquito midgut

Article

Author: Li, Meilin ; Zhou, Taoli ; Huang, Wei ; Liu, Taiping ; Jiang, Lubin ; Tan, Nie ; Gao, Yuanli ; Xu, Wenyue ; Li, Jian ; Guo, Shuai ; Jiao, Shiming ; Jiao, Zhiwei ; Lin, Zurui ; Zhang, Jian ; Li, Jianyong ; He, Biao ; Fan, Yongling ; Zhu, Feng ; Wang, Sibao

Host-derived factors ingested during mosquito blood feeding are poorly understood modulators of malaria transmission. Here, we demonstrated that host complement C3, acquired by mosquitoes during Plasmodium infection, significantly enhanced rodent malaria infection in laboratory-reared mosquitoes. This effect was recapitulated in field-caught Anopheles sinensis mosquitoes, confirming its relevance to malaria transmission in a more natural setting. Moreover, host-derived C3 significantly reduced the efficacy of anti-Pfs25 antibodies in blocking malaria transmission. Mechanistically, host-derived C3 lyses the mosquito midgut symbiont Elizabethkingia anophelis ( E. anophelis )—a bacterium that intrinsically suppresses parasite development by blocking the zygote-to-ookinete transition. Strikingly, host-derived C3 in mosquitoes appears to be activated by the alternative pathway, and inhibiting Factor B with Iptacopan (LNP023) reduced Plasmodium falciparum ( P. falciparum ) infection, while increased the efficacy of anti-Pfs25 antibodies to blocking P. falciparum transmission in the standard membrane-feeding assay. Therefore, this study describes a strategy of the malaria parasite to utilize host complement C3 to promote its transmission and provides us with an avenue to block malaria transmission and improve the blocking efficacy of anti-Pfs25 antibodies by the inhibition of C3 activation.

01 Jun 2025·EJHaem

Effectiveness of Iptacopan Versus C5 Inhibitors in Complement Inhibitor‐Naive Patients With Paroxysmal Nocturnal Haemoglobinuria

Article

Author: Fermont, Jilles M. ; Brindel, Isabelle ; Kelly, Richard J. ; Holt, Matthew ; Dahlke, Marion ; de Fontbrune, Flore Sicre ; Ansari, Soudeh ; Maafa, Lynda ; de Latour, Régis Peffault

ABSTRACT:

Background:

Paroxysmal nocturnal haemoglobinuria (PNH) is characterised by haemolytic anaemia, bone marrow failure and thrombosis. The single‐arm phase 3 APPOINT‐PNH trial (NCT04820530) investigating iptacopan monotherapy in complement inhibitor‐naive patients demonstrated significant haemoglobin concentration improvements.

Methods:

We used target trial emulation to retrospectively predict outcomes if APPOINT‐PNH trial patients had received C5 inhibitors instead of iptacopan. Estimates were derived from the real‐world APPEX cohort treated with routine C5 inhibitors. The study used benchmarking and comparative effectiveness to evaluate the haematological response in APPOINT‐PNH if patients had received C5 inhibitors. Treatment effect was estimated using propensity scores to model the probability of trial inclusion based on baseline covariates, followed by fitting an outcome model to the APPEX cohort.

Results:

The analysis of 125 patients showed all estimated treatment effects (95% confidence interval) favoured iptacopan over C5 inhibitors: differences in the proportion of patients achieving haemoglobin increase from baseline of ≥ 2 g/dL, 68.2% (40.9–95.6); haemoglobin levels of ≥ 12 g/dL, 53.4% (31.4–75.3); transfusion independence, 38.8% (15.1–62.5); ratio of percent change from baseline in lactate dehydrogenase levels, 0.51 (0.40–0.67); change from baseline in reticulocytes, −75.5 × 109/L (−106.9, −44.2).

Conclusions:

Results indicate C5 inhibitor‐naive patients with PNH may experience greater haematological response with iptacopan than with C5 inhibitors.

Trial Registration:

ClinicalTrials.gov identifier: NCT05842486

01 Jun 2025·Lancet Haematology

Oral iptacopan monotherapy in paroxysmal nocturnal haemoglobinuria: final 48-week results from the open-label, randomised, phase 3 APPLY-PNH trial in anti-C5-treated patients and the open-label, single-arm, phase 3 APPOINT-PNH trial in patients previously untreated with complement inhibitors

Article

Author: Griffin, Morag ; Mauad, Vitor A Q ; Yang, Chen ; Di Bona, Eros ; Langemeijer, Saskia M C ; Uchiyama, Michihiro ; Maitra, Samopriyo ; Panse, Jens ; Pullarkat, Vinod ; Röth, Alexander ; Maciejewski, Jaroslaw P ; Barcellini, Wilma ; Marano, Luana ; Ferber, Philippe ; Gandhi, Shreyans ; Risitano, Antonio M ; Han, Bing ; Kitawaki, Toshio ; Höchsmann, Britta ; Solar-Yohay, Susan ; Ueda, Yasutaka ; Lee, Lily Wong Lee ; Kutlar, Abdullah ; Mahajan, Navin ; Tavitian, Suzanne ; Chew, Lee Ping ; Zhang, Li ; Jang, Jun Ho ; Frieri, Camilla ; Li, Shujie ; Schafhausen, Philippe ; Dahlke, Marion ; Kelly, Richard J ; Beggiato, Eloise ; de Latour, Régis Peffault ; Terriou, Louis ; Kulasekararaj, Austin G ; Schubert, Jörg ; Trikha, Roochi ; Fu, Rong ; Huang, Wei-Han ; Gaya, Anna ; Liu, Hui ; de Castro, Carlos M ; Yap, Eng-Soo ; Thorburn, Christine ; Verles, Aurelie ; de Fontbrune, Flore Sicre ; Wang, Zhixin ; Scheinberg, Phillip ; Kerloëguen, Cécile ; Schrezenmeier, Hubert ; Lawniczek, Tomasz ; Kumar, Rakesh ; Notaro, Rosario ; Alashkar, Ferras

BACKGROUND:

The factor B inhibitor iptacopan improved 24-week outcomes in adult patients with paroxysmal nocturnal haemoglobinuria in the phase 3 APPLY-PNH and APPOINT-PNH trials; the trial extension periods assessed clinical activity and safety up to 48 weeks. Here, we report the final 48-week data from APPLY-PNH and APPOINT-PNH.

METHODS:

In both APPLY-PNH and APPOINT-PNH trials, patients were aged 18 years or older, with paroxysmal nocturnal haemoglobinuria (red and white blood cell population sizes ≥10%) and without laboratory evidence of bone marrow failure. In APPLY-PNH (an open-label, randomised, phase 3 trial conducted in 39 centres [38 hospitals, one outpatient research clinic] from 12 countries or regions), patients with haemoglobin concentration lower than 10 g/dL on anti-C5 treatment (stable eculizumab or ravulizumab regimen for ≥6 months) were randomly assigned (8:5) via interactive response technology to either receive oral iptacopan 200 mg twice daily (iptacopan group) or to continue their individual intravenous eculizumab or ravulizumab regimen for 24 weeks (anti-C5 group). Randomisation was stratified by type of anti-C5 and receipt of red blood cell (RBC) transfusions in the preceding 6 months. In APPOINT-PNH (an open-label, single-arm, phase 3 trial conducted in 12 hospitals from eight countries), complement inhibitor-naive patients with paroxysmal nocturnal haemoglobinuria and with haemoglobin concentration lower than 10 g/dL and lactate dehydrogenase (LDH) concentration higher than 1·5 times the upper limit of normal received iptacopan 200 mg twice daily for 24 weeks. Both trials had 24-week extension periods in which all patients received iptacopan monotherapy. Primary endpoints were the proportion of patients with an increase from baseline in haemoglobin concentration of 2 g/dL or higher (APPLY-PNH and APPOINT-PNH) and haemoglobin concentration 12 g/dL or higher (APPLY-PNH) between weeks 18 and 24, all in the absence of RBC transfusions between weeks 2 and 24; results for these primary endpoints have been reported previously. We report final activity and safety data at the completion of both trials (week 48). Prespecified endpoints at week 48 included percentage of patients with a haemoglobin increase from baseline of 2 g/dL or higher or haemoglobin 12 g/dL or higher (including post-transfusion data). Efficacy data were analysed per the intention-to-treat principle, and safety was analysed according to the treatment that patients received. APPLY-PNH and APPOINT-PNH are registered with ClinicalTrials.gov, NCT04558918 and NCT04820530, respectively.

FINDINGS:

In APPLY-PNH, between Jan 25, 2021, and April 8, 2022, 62 patients (43 [69%] female, 19 [31%] male; 48 [77%] White, 12 [19%] Asian, two [3%] Black) were randomly assigned to the iptacopan group and 35 patients (24 [69%] female, 11 [31%] male; 26 [74%] White, seven [20%] Asian, two [6%] Black) to the anti-C5 group; 61 (98%) and 34 (97%), respectively, entered the extension period. At trial completion (March 6, 2023), the median duration of iptacopan treatment was 337 days (IQR 168-338). In APPOINT-PNH, 40 patients were enrolled between July 19, 2021, and May 17, 2022, and received iptacopan (17 [43%] female, 23 [58%] male; 12 [30%] White, 27 [68%] Asian, one [3%] Black); all entered the extension period. At trial completion (April 18, 2023), the median duration of iptacopan treatment was 337 days (IQR 337-344). At week 48, irrespective of RBC transfusions, the number of patients who had an increase in haemoglobin concentration of 2 g/dL or higher was 51 (86%) of 59 in the APPLY-PNH iptacopan group, 21 (72%) of 29 in the APPLY-PNH anti-C5-to-iptacopan group, and 38 (97%) of 39 in APPOINT-PNH. The number of patients who had haemoglobin concentration of 12 g/dL or higher at week 48 was 40 (68%) of 59 in the APPLY-PNH iptacopan group, 17 (59%) of 29 in the APPLY-PNH anti-C5-to-iptacopan group, and 31 (79%) of 39 in APPOINT-PNH. There were no treatment discontinuations because of treatment-emergent adverse events or deaths. Across the 48-week trials, clinical breakthrough haemolysis occurred in seven (7%) of 96 iptacopan-treated patients in APPLY-PNH (including both groups) and two (5%) of 40 in APPOINT-PNH, but it was generally mild or moderate with no iptacopan discontinuation. Three major adverse vascular events occurred in APPLY-PNH by trial completion; all were considered unrelated to iptacopan. The most common treatment-emergent adverse event was COVID-19 in APPLY-PNH (iptacopan: 18/62 patients [29%]; anti-C5-to-iptacopan: 8/34 [24%]) and headache in APPOINT-PNH (12/40 [30%]). Severe and serious treatment-emergent adverse events were experienced by six (10%) and nine (15%) of 62 patients in the APPLY-PNH iptacopan group, respectively; in APPOINT-PNH, these were experienced by four (10%) and eight (20%) of 40 patients, respectively. The most common serious treatment-emergent adverse event was COVID-19, occurring in one (2%) of 62 patients in the APPLY-PNH iptacopan group and two (5%) of 40 patients in APPOINT-PNH. No severe treatment-emergent adverse events occurred in more than one patient.

INTERPRETATION:

Long-term data indicate durable haemolysis control with iptacopan in paroxysmal nocturnal haemoglobinuria, maintained normal or near-normal haemoglobin, and no new safety concerns. We believe that these data support iptacopan as a potential therapy option, suggesting that we are in a new treatment era for paroxysmal nocturnal haemoglobinuria.

FUNDING:

Novartis.

216

News (Medical) associated with Iptacopan

12 Jun 2025

·pipelinereview.com

Novartis Fabhalta® shows statistically significant and clinically meaningful improvements in hemoglobin in new population of patients with PNH

BASEL, Switzerland I June 12, 2025 I Novartis announced positive 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) with Hb levels ≥10g/dL who switched from anti-C5 therapies (eculizumab or ravulizumab) 1 . After 24 weeks of treatment with Fabhalta, the Hb level improved on average by 2.01 g/dL (95% CI, 1.74, 2.29) with most patients achieving normal or near-normal levels 1 . Data will be presented at the European Hematology Association (EHA) Congress 2025. “Today, some patients living with PNH have unmet needs not addressed by eculizumab or ravulizumab,” said Austin Kulasekararaj, Consultant Hematologist, Kings College Hospital and Kings College London. “The positive results from APPULSE-PNH reinforce that Fabhalta can provide clinically meaningful improvements in hemoglobin among patients with higher baseline hemoglobin levels than those enrolled in previous trials, while offering an oral monotherapy for patients.” No patients required transfusion during the study, and the vast majority (92.7%) achieved Hb ≥12g/dL, reaching normal or near-normal levels 1 . Patients treated with Fabhalta also reported clinically meaningful improvements in fatigue (as measured by FACIT-Fatigue score) through Day 168, reaching absolute levels similar to those reported in the general population 1,3,4 . Furthermore, patients treated with Fabhalta maintained intravascular hemolysis control and resolved extravascular hemolysis control, as demonstrated by lactate dehydrogenase levels (<1.5 upper limit of normal) and a reduction in absolute reticulocyte count 1 . “Novartis is dedicated to advancing research and innovation that can transform care and significantly improve the lives of people living with PNH and those who support them,” said Shreeram Aradhye, M.D., President, Development and Chief Medical Officer, Novartis. “New data from APPULSE-PNH, combined with findings from the Phase III roll-over extension of the APPLY-PNH and APPOINT-PNH studies, reinforce the efficacy and safety profile of Fabhalta in delivering real benefits to patients. Fabhalta is the first and only oral monotherapy currently available for the treatment of adults with PNH, regardless of previous treatment experience.” Alongside APPULSE-PNH, longer-term data from patients initially included in the APPLY-PNH and APPOINT-PNH Phase III studies will be presented at EHA 1 . In APPULSE-PNH and the Phase III roll-over extension study of the APPLY-PNH and APPOINT-PNH studies, Fabhalta was well-tolerated with no new safety signals, consistent with previously reported data 1,5 . About paroxysmal nocturnal hemoglobinuria (PNH) PNH is a rare, chronic and serious complement-mediated blood disorder 6 . 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 system 6,7 . 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 symptoms 6,7 . It is estimated that approximately 10-20 people per million worldwide live with PNH 6 . Although PNH can develop at any age, it is often diagnosed in people between 30-40 years old 8,9 . 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 hours 10 . Despite treatment with anti-C5 therapies, a large proportion of people with PNH remain anemic, and some dependent on blood transfusions 7,11-16 . About APPULSE-PNH APPULSE-PNH (NCT05630001) is a Phase IIIB, multinational, 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 weeks 2 . 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 period 2,17 . The primary endpoint is change from baseline Hb levels after 24 weeks of treatment with Fabhalta 2,17 . About APPLY-PNH APPLY-PNH (NCT04558918) is a Phase III, randomized, multinational, multicenter, active-comparator controlled, open-label trial to evaluate the efficacy and safety of twice-daily, oral Fabhalta ® (iptacopan) monotherapy (200mg) for the treatment of PNH by demonstrating the superiority of Fabhalta compared to anti-C5 therapies (eculizumab or ravulizumab) in adult patients presenting with residual anemia (Hb <10 g/dl) despite a stable regimen of anti-C5 treatment in the last six months prior to randomization 5,18 . About APPOINT-PNH APPOINT-PNH (NCT04820530) is a Phase III, multinational, multicenter, open-label, single-arm study to evaluate the efficacy and safety of twice-daily, oral Fabhalta ® (iptacopan) monotherapy (200mg) in adult PNH patients who are naïve to complement inhibitor therapy, including anti-C5 therapies (eculizumab or ravulizumab) 19,20 . About Fabhalta® (iptacopan) Fabhalta (iptacopan) is an oral, Factor B inhibitor of the alternative complement pathway 21,22 . 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) and accelerated approval in the US in August 2024 for the reduction of proteinuria in adults with primary IgA nephropathy (IgAN) at risk of rapid disease progression (generally UPCR ≥1.5 g/g 1.5 g/g) 1,23,24 . In 2025, Fabhalta received FDA and EC approval for the treatment of adults with C3 glomerulopathy (C3G) to reduce proteinuria, making it the first and only treatment approved for this condition 25-31 . 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). Studies are ongoing to evaluate the safety and efficacy profiles in these investigational indications and support potential regulatory submissions 32-35 . 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 . References SOURCE: Novartis

Phase 3Clinical ResultDrug Approval

12 Jun 2025

·firstwordpharma.com

Novartis makes strong case for Fabhalta in patients switching from anti-C5 PNH therapies

Novartis unveiled detailed findings from a third late-stage study of its oral complement inhibitor Fabhalta (iptacopan) in paroxysmal nocturnal haemoglobinuria (PNH), demonstrating notable clinical benefits when switching from anti-C5 therapies to twice-daily Fabhalta monotherapy. Results from the 52-participant Phase IIIB APPULSE-PNH study top-lined in December last year, showed that average haemoglobin (Hb) levels increased from baseline after 24 weeks of Fabhalta monotherapy in patients on a stable regimen of anti-C5 therapies — AstraZeneca’s Soliris (eculizumab) or Ultomiris (ravulizumab) — for ≥6 months before screening, with baseline Hb ≥10 g/dL and no transfusions during that period.New data released on Thursday ahead of the European Haematology Association (EHA) Congress, showed that switching from anti-C5 therapies to Fabhalta led to an average Hb increase of 2.01 g/dL, with 92.7% of patients reaching normal or near-normal levels at 24 weeks. Notably, no patients required transfusion during the study period.Fabhalta recipients also reported significant improvements in fatigue on the FACIT-Fatigue scores by day 168, reaching absolute levels seen in the general population. Moreover, the treatment maintained intravascular haemolysis control whilst resolving extravascular haemolysis, demonstrated through sustained lactate dehydrogenase levels below 1.5 times the upper limit of normal and reduced absolute reticulocyte counts.Safety-wise, the drug was well-tolerated, with no new signals identified, consistent with previous studies.The results build on the earlier Phase III trials — APPLY-PNH and APPOINT-PNH in previously treated patients and the complement inhibitor-naïve patients, respectively, which supported Fabhalta’s approval for indication in the US and EU. “New data from APPULSE-PNH, combined with findings from…APPLY-PNH and APPOINT-PNH studies, reinforce the efficacy and safety profile of Fabhalta in delivering real benefits to patients," said Shreeram Aradhye, chief medical officer at Novartis.Besides PNH, Fabhalta also holds FDA approvals for IgA nephropathy and complement 3 glomerulopathy.

Clinical ResultPhase 3Drug Approval

09 Jun 2025

·www.einpresswire.com

Atypical Hemolytic Uremic Syndrome (aHUS): Breaking the Boundaries of Traditional Treatment | Competitive Intelligence

Next-gen aHUS treatments are shifting from IV infusions to oral and subcutaneous options-offering new hope in safety, efficacy, and patient ease. The aHUS therapy landscape is on the cusp of disruption-with emerging oral drugs and biosimilars reshaping market dynamics and patient access.” — DataM Intelligence AUSTIN, TX, UNITED STATES, June 9, 2025 / EINPresswire.com / -- Atypical hemolytic uremic syndrome (aHUS) is a rare, life-threatening condition that has challenged clinicians for decades due to its complex pathophysiology and unpredictable relapses. Primarily caused by uncontrolled activation of the complement system-a vital part of immune defense-this disorder results in the body mistakenly attacking its own endothelial cells. This autoimmune-like attack leads to blood clots in small vessels, hemolytic anemia, low platelets, and acute kidney injury. The cascade of events often unfolds rapidly, leaving minimal room for therapeutic delay. Book Your Free CI Consultation Call: https://www.datamintelligence.com/strategic-insights/ci/atypical-hemolytic-uremic-syndrome-ahus Historically, the clinical landscape for aHUS treatment was limited and reactive. However, the emergence of complement inhibitors such as Soliris (eculizumab) and Ultomiris (ravulizumab) has dramatically transformed patient outcomes. These targeted therapies block the complement component C5, thereby halting the terminal pathway responsible for endothelial injury. Despite their efficacy, these treatments bring challenges-namely, high costs, intravenous dosing burdens, and long-term immunosuppression. Epidemiology and Disease Burden Though rare, aHUS is now recognized more frequently due to improved diagnostic capabilities and awareness. Incidence estimates suggest approximately 1 to 2 cases per million population annually, with a higher prevalence in pediatric patients and young adults. Despite being uncommon, the economic and clinical burden is significant. Frequent hospitalizations, renal replacement therapies, and lifelong medication usage make it a resource-intensive condition for both patients and health systems. Looking ahead, as diagnosis rates continue to rise and access to therapy improves globally, the treated patient population is expected to expand steadily over the next decade. Market Leaders: Soliris and Ultomiris Soliris, the first-in-class anti-C5 monoclonal antibody, has long been the backbone of aHUS management. Delivered via intravenous infusion every two weeks, it provides rapid disease control and kidney function stabilization. Ultomiris, a long-acting version of Soliris, offers dosing every eight weeks and has quickly gained traction due to its extended half-life and comparable efficacy. While both drugs offer life-altering benefits, their IV delivery model and high cost present logistical and economic hurdles-particularly in resource-limited settings. Furthermore, patients on chronic complement inhibition face elevated risks of infections, especially from encapsulated organisms like Neisseria meningitidis, requiring mandatory vaccination and close monitoring. Biosimilars Enter the Arena: Expanding Access To address cost constraints, biosimilars such as Bkemv and EPYSQLI have recently entered the market. Both mirror the efficacy and safety profile of Soliris but at reduced prices. This has significant implications for healthcare systems seeking cost-effective treatment options and for patients in emerging markets where branded biologics were previously out of reach. The entry of biosimilars is expected to trigger pricing pressure on originator brands and encourage broader adoption of complement blockade therapy in previously underserved regions. Emerging Innovations: Convenience Redefined The next wave of innovation in aHUS centers around patient-centric improvements-most notably the shift from intravenous to oral or subcutaneous delivery. Fabhalta (Iptacopan), an oral complement factor B inhibitor, represents a potentially game-changing option. By targeting the alternative pathway upstream of C5, Fabhalta may offer broader complement control. Its oral daily dosing also addresses the burden of frequent infusions, enhancing quality of life and adherence. Another frontrunner is Crovalimab (Piasky), a subcutaneous anti-C5 monoclonal antibody recently approved in several regions. Its monthly SC administration empowers patients with greater autonomy and reduces hospital visits-especially appealing for younger, working patients or those in remote areas. These innovations are not merely incremental-they have the potential to disrupt the current therapeutic paradigm, offering alternatives that align more closely with patient preferences. Pipeline Outlook: What’s Next? The aHUS pipeline includes promising assets in various stages of development, including novel monoclonal antibodies, small molecules, and even RNA interference (RNAi) therapies. These next-gen modalities aim to deliver differentiated value-whether through new mechanisms of action, improved safety profiles, or enhanced pharmacokinetics. Key targets include: - Complement factor B and C3, which offer upstream inhibition for more comprehensive control. - Gene therapies under preclinical evaluation, aiming for long-term or potentially curative outcomes. - Oral agents and self-administered injectables, reducing the logistical barriers of hospital-based care. While Soliris and Ultomiris set a high bar for efficacy and safety, pipeline therapies must offer clear advantages-either in administration, tolerability, or cost-effectiveness-to capture significant market share. Target Opportunity Profile (TOP): Where the Gaps Lie To secure a strong foothold in the evolving aHUS market, developers must address the following unmet needs: - Convenience: Intravenous dosing every two to eight weeks limits patient freedom. Oral or subcutaneous routes are increasingly preferred. - Affordability: The high cost of branded therapies restricts global access. Cost-effective alternatives or pricing models will be key. - Safety: While current therapies are generally well tolerated, they carry infection risks. Novel agents must aim to reduce immunosuppression-related complications. - Innovation in Mechanism: Moving beyond C5 to upstream targets may offer added efficacy in difficult or resistant cases. - Real-World Outcomes: Long-term data on kidney preservation, survival, and quality of life will influence payer decisions and treatment guidelines. Competitive Dynamics: A Market in Transition The market for aHUS treatment is no longer defined solely by biologics delivered via infusion centers. With oral small molecules like Fabhalta and subcutaneous agents like Crovalimab gaining traction, the competitive landscape is undergoing rapid transformation. Manufacturers must now compete not only on efficacy but also on convenience, safety, price, and patient experience. Biosimilars are squeezing margins while expanding access. Meanwhile, pipeline entrants promise to reset expectations entirely-with oral, gene-based, and potentially curative approaches in development. Download Free Sample Report: https://www.datamintelligence.com/strategic-insights/sample/atypical-hemolytic-uremic-syndrome-ahus Conclusion The treatment of atypical hemolytic uremic syndrome has come a long way-from managing life-threatening relapses with supportive care to providing targeted, disease-modifying therapy. But the next frontier is patient-centric: offering safe, effective, and affordable treatment that fits seamlessly into patients’ lives. Oral drugs like Fabhalta and injectables like Crovalimab mark a pivotal shift, challenging the status quo and opening the door to more personalized and accessible care. As biosimilars democratize access and innovation drives differentiation, the aHUS market is poised for significant evolution in the years ahead. Read Related CI Reports: 1. Eosinophilic Esophagitis | Competitive Intelligence 2. Warm Autoimmune Hemolytic Anemia | CI Insights Sai Kumar DataM Intelligence 4market Research LLP +1 877-441-4866 email us here Visit us on social media: LinkedIn X Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.

Drug Approval

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
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.

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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	Germany	Novartis Pharma AG	31 Jul 2024
Myasthenia Gravis	Phase 3	Greece	Novartis Pharmaceuticals Canada, Inc.	31 Jul 2024
Myasthenia Gravis	Phase 3	Greece	Novartis Pharma AG	31 Jul 2024

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04817618 (APPEAR-C3G, FDA_CDER) Manual	Phase 3	C3 glomerulopathy	74	FABHALTA 200 mg	ccjkqpzgrl(blwtdjraco) = ksqvvikffc wxrbgvxrpg (gmihksfrop, 0.57 - 0.85) View more	Positive	20 Mar 2025
				Placebo	ccjkqpzgrl(blwtdjraco) = mrgspjkvdc wxrbgvxrpg (gmihksfrop, 0.88 - 1.31) View more
NCT04820530 \| NCT04558918 (APPOINT-PNH, Pubmed) Manual	Phase 3	Hemoglobinuria, Paroxysmal	135	iptacopan (C5i-experienced patients)	zdcwjqmzzv(oaalczxkhf) = mxneaepobj qmxoqscfpk (inoahgojkl ) View more	Positive	07 Jan 2025
				Placebo (C5i-experienced patients)	zdcwjqmzzv(oaalczxkhf) = dcqcmierpb qmxoqscfpk (inoahgojkl ) View more
ASH2024	Not Applicable	Hemoglobinuria, Paroxysmal	-	Iptacopan monotherapy 200 mg twice daily	imsxbwvabf(mpbcuybzqe) = evubfqfocl jsxwusqkvj (ydcwnfddhp, 87.4) View more	-	09 Dec 2024
				Eculizumab	imsxbwvabf(mpbcuybzqe) = qyggroocmk jsxwusqkvj (ydcwnfddhp, 77.2) View more
NCT05086744 (ASH2024)	Phase 2	Cold Agglutinin Disease	-	Iptacopan 200 mg	nvlxlznapd(frbohqmpyv) = 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. oxqvqtcfvu (urypogiwgj )	-	08 Dec 2024
NCT04578834 (APPLAUSE-IgAN, FDA_CDER) Manual	Phase 3	Glomerulonephritis, IGA	250	FABHALTA	vnbzlnkjra(bjnzhpcrvu) = fiwslfygis hnvvxvkyyx (hsweleigzj, 36 - 51) View more	Positive	07 Aug 2024
				Placebo	vnbzlnkjra(bjnzhpcrvu) = mlpfyxrdpg hnvvxvkyyx (hsweleigzj, -5 to 21) View more
NCT04154787 (CTgov)	Phase 2	Idiopathic Membranous Glomerulonephritis	37	Rituximab	wfsxpupvei(akoczknpjz) = tfbbofsybu hcdngpkark (tqownswcdt, lvekzfqhpn - wieuikvoxj) View more	-	05 Jul 2024
NCT04817618 (APPEAR-C3G, PRNewswire 1 \| PRNewswire 2) Manual	Phase 3	C3 glomerulopathy	-	Fabhalta (iptacopan)	ouixfgefzw(lucaxjrrzr) = zupftikbys medkmjodbf (mynrfrhiqn )	Positive	25 May 2024
				Placebo	-
NCT04820530 (APPOINT-PNH, EHA2024) Manual	Phase 3	Hemoglobinuria, Paroxysmal	40	Iptacopan 200 mg	nwoqozowdy(ladheyzguv) = imrqrtxzol jtohegmzvg (sfsojktwqm ) View more	Positive	14 May 2024
NCT04558918 \| NCT03500549 (APPLY-PNH \| PEGASUS, EHA2024) Manual	Phase 3	Hemoglobinuria, Paroxysmal Maintenance	103	Iptacopan	itbsdqxwpi(bezaxzmjcy) = vrwifyxxph gbgkitiesp (gzumqlbxso ) View more	Positive	14 May 2024
				Pegcetacoplan	itbsdqxwpi(bezaxzmjcy) = mmjdnclmab gbgkitiesp (gzumqlbxso )
NCT04558918 (APPLY-PNH, EHA2024)	Phase 3	Hemoglobinuria, Paroxysmal C3 fragment deposition	95	Iptacopan 200 mg twice daily	iiqfeabpyf(buwhhhpcdf) = gxtxeknkfw adalyltxaf (xtnpuqpdsv, -16.1%) View more	Positive	14 May 2024
				Anti-C5 inhibitors	iiqfeabpyf(buwhhhpcdf) = tnyfjspzcg adalyltxaf (xtnpuqpdsv, -16.9%) View more

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