Latest news with #Fabhalta
Yahoo
08-07-2025
- Health
- Yahoo
Market Barriers Continue to Challenge PNH Treatment Optimization Despite Emerging Options, According to Spherix Global Insights
New research highlights significant insurance-driven hurdles, increasing second-line uptake of Fabhalta (Novartis), and strong physician interest in pipeline therapies including zaltenibart (Omeros), pozelimab-cemdisiran (Regeneron/Alnylam), and ruxaprubart (NovelMed). EXTON, PA, July 08, 2025 (GLOBE NEWSWIRE) -- Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, chronic blood disorder that can affect individuals of any age, race, or gender, though it is most commonly diagnosed in adults in their 30s and 40s. In the United States, an estimated 400 to 500 new cases are identified annually. The clinical presentation of PNH varies widely—some patients experience only mild symptoms, while others face life-threatening complications requiring interventions such as immunosuppressive therapy or blood transfusions. ¹ Hematologists are actively adapting their prescribing strategies in paroxysmal nocturnal hemoglobinuria (PNH), with future pipeline entrants poised to further reshape the treatment landscape. According to Spherix Global Insights' Q2 2025 update from RealTime Dynamix™: PNH (US), the availability and adoption of biosimilars are expected to significantly expand the overall treated PNH population—bringing more patients onto complement inhibitor therapy than ever before. The Spherix research projects that biosimilar eculizumab products—Bkemv (Amgen) and Epysqli (Teva/Samsung Bioepis)—will continue to gain traction, capturing share from both branded Soliris and Ultomiris (Alexion/AstraZeneca), as hematologists grow increasingly confident in the clinical utility and cost-effectiveness of biosimilar alternatives. Use of novel entrants in the second line is growing as well, with therapies such as Fabhalta (Novaratis) and Voydeya (AstraZeneca) capturing an increasing share of recent prescribing. These agents are selected for patients who progress or are sub-optimally controlled on C5 inhibitors. Despite these gains, access remains a major commercial hurdle. Many patients with PNH are believed to be suboptimally managed due to persistent insurance-related barriers. Nearly all prescribers report significant challenges navigating the prior authorization process, citing inconsistent payer requirements, delays in drug procurement, and a growing reliance on artificial intelligence by insurers to further restrict coverage. This evolving dynamic has become a major source of frustration for hematologists, often delaying timely access to appropriate therapies and complicating clinical decision-making. The competitive landscape in PNH continues to intensify, with future therapies gaining interest among both academic and community hematologists. The latest Spherix study incorporates target product profiles and prescriber expectations for emerging agents, including zaltenibart (Omeros), a MASP-3 inhibitor that has captured strong physician interest. Most hematologists anticipate zaltenibart will be adopted alongside existing first- and second-line therapies upon approval, driven by high efficacy expectations and its potential to address persistent unmet needs. Other late-stage candidates—such as pozelimab-cemdisiran (Regeneron/Alnylam) and ruxaprubart (NovelMed)—also register meaningful commercial and clinical potential among surveyed specialists. 'These results underscore that physicians aren't just passively watching the market evolve – they're preparing to act,' said Sarah Hendry, Hematology Franchise Head at Spherix. 'With rising expectations for access support and guideline alignment, manufacturers that combine clinical value with commercial enablement will be best positioned for success.' As was seen in Paris at the IPIG international conference when Spherix presented data from their Patient Chart Dynamix™, membership in critical professional societies is growing as hematologists seek the guidance in PNH treatments and care. The most recent release offers a comprehensive look at the U.S. PNH market, including prescribing patterns, brand dynamics, access challenges, and future projections. Delivered quarterly, RealTime Dynamix™ helps commercial teams anticipate shifts, benchmark against competitors, and support strategic planning ahead of key market events. RealTime Dynamix™ is an independent service providing strategic guidance through quarterly or semiannual reports, which include market trending and a fresh infusion of event-driven and variable content with each wave. The reports provide an unbiased view of the competitive landscape within rapidly evolving specialty markets, fueled by robust HCP primary research and our in-house team of experts. Patient Chart Dynamix™ is an independent service that includes robust patient chart audits and integrated specialist surveys fielded biannually. This research provides an in-depth, real-world view of treatment practices by combining verified patient data with attitudinal insights from physicians. The series highlights clinical decision-making, treatment sequencing, and outcomes for targeted patient populations across key therapeutic areas. About Spherix Global Insights Spherix is a leading independent market intelligence and advisory firm that delivers commercial value to the global life sciences industry, across the brand lifecycle. The seasoned team of Spherix experts provides an unbiased and holistic view of the landscape within rapidly evolving specialty markets, including dermatology, gastroenterology, rheumatology, nephrology, neurology, ophthalmology, and hematology. Spherix clients stay ahead of the curve with the perspective of the extensive Spherix Physician Community. As a trusted advisor and industry thought leader, Spherix's unparalleled market insights and advisory services empower clients to make better decisions and unlock opportunities for growth. To learn more about Spherix Global Insights, visit or connect through LinkedIn. For more details on Spherix's primary market research reports and interactive dashboard offerings, visit or register here: NOTICE: All company, brand or product names in this press release are trademarks of their respective holders. The findings and opinions expressed within are based on Spherix Global Insight's analysis and do not imply a relationship with or endorsement of the companies or brands mentioned in this press release. CONTACT: Sarah Hendry, Hematology Franchise Head Spherix Global Insights 4848794284 in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
16-06-2025
- Business
- Yahoo
Berenberg Bank Maintains a Hold Rating on Novartis (NVS)
Novartis AG (NYSE:NVS) is one of the 11 Best Drug Stocks to Buy According to Hedge Funds. On June 12, Berenberg Bank analyst Luisa Hector maintained a Hold rating on Novartis AG (NYSE:NVS) and set a price target of CHF89.00. The same day, the company reported statistically significant improvements in the Phase IIIB APPULSE-PNH study. The study demonstrated clinically meaningful improvements in hemoglobin (Hb) levels of 2.01 g/dL on average in adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who switched to Fabhalta. A doctor holding a microscope in front of a laboratory sample of healthcare products. None of the patients treated with Fabhalta required transfusions, experienced major adverse vascular events during the treatment period, or experienced breakthrough hemolysis (BTH). Novartis AG (NYSE:NVS) develops, manufactures, and markets healthcare products. The company operates through the Innovative Medicines, Sandoz, and Corporate segments. It is the tenth-best drug stock to invest in now and is headquartered in Basel, Switzerland. While we acknowledge the potential of NVS as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. READ NEXT: The Best and Worst Dow Stocks for the Next 12 Months and 10 Unstoppable Stocks That Could Double Your Money. Disclosure: None. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
13-06-2025
- Health
- Yahoo
Novartis reports positive results from Phase IIIB study of iptacopan for PNH
Novartis has reported positive outcomes from the multi-centre Phase IIIB APPULSE-PNH study of oral Fabhalta (iptacopan) in adults with paroxysmal nocturnal haemoglobinuria (PNH). These findings are set to be presented at the European Hematology Association (EHA) Congress in 2025. The single-arm, open-label, multinational trial assessed the oral therapy in PNH subjects with haemoglobin (Hb) levels of ≥10g/dL, who transitioned from anti-C5 therapies (eculizumab or ravulizumab). It enrolled 52 subjects and aimed to expand the clinical evidence for the therapy by including patients with higher baseline Hb levels than those in the pivotal Phase III programme. Following 24 weeks of treatment with the therapy, subjects experienced an average increase in Hb levels of 2.01g/dL, with a significant number achieving normal or near-normal levels. During the trial, none of the participants needed blood transfusions, and 92.7% of them reached Hb levels of at least 12g/dL. In addition, subjects reported improvements in fatigue, with Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scores aligning with those of the general population by day 168. Novartis noted that Fabhalta-treated subjects also maintained intravascular haemolysis control and resolved extravascular haemolysis control, indicated by lactate dehydrogenase levels remaining below 1.5 times the upper limit of normal and a decrease in absolute reticulocyte count. Along with these findings, the company will also present longer-term data from the APPLY-PNH and APPOINT-PNH Phase III trials at the EHA Congress. Novartis development president and chief medical officer Shreeram Aradhye said: '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 was discovered by Novartis and acts as a Factor B inhibitor of the alternative complement pathway. It also offers a new treatment option for the rare and life-threatening complement-mediated blood disorder, PNH. The company recently shared topline outcomes from the Phase III PSMAddition trial's pre-specified interim analysis, in which Pluvicto plus standard of care (SoC) treatment demonstrated a benefit in treating prostate-specific membrane antigen (PSMA)-positive metastatic hormone-sensitive prostate cancer (mHSPC). "Novartis reports positive results from Phase IIIB study of iptacopan for PNH" was originally created and published by Clinical Trials Arena, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Sign in to access your portfolio
Business Upturn
12-06-2025
- Health
- Business Upturn
Novartis Fabhalta® shows statistically significant and clinically meaningful improvements in hemoglobin in new population of patients with PNH
By GlobeNewswire Published on June 12, 2025, 11:00 IST In the Phase IIIB APPULSE-PNH study, adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who switched to Fabhalta experienced clinically meaningful improvements in hemoglobin (Hb) levels of 2.01 g/dL on average 1,2 APPULSE-PNH evaluated Fabhalta in a population with higher baseline hemoglobin (Hb) levels than those enrolled in the pivotal Phase III program, expanding the clinical evidence base 1 ,2 No patients treated with Fabhalta required transfusions, experienced breakthrough hemolysis (BTH) or had any major adverse vascular events during the treatment period 1 Patients treated with Fabhalta reported an average improvement in fatigue from baseline of 4.88 points at Day 84 and 4.29 points at Day 168 of the study (as measured by FACIT-Fatigue score) 1 Basel, June 12, 2025 – 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 levels1. 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 population1,3,4. 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 count1. '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 EHA1. 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 data1,5. About paroxysmal nocturnal hemoglobinuria (PNH) PNH is a rare, chronic and serious complement-mediated blood disorder6. 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 system6,7. 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 symptoms6,7. It is estimated that approximately 10-20 people per million worldwide live with PNH6. Although PNH can develop at any age, it is often diagnosed in people between 30-40 years old8,9. 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 hours10. Despite treatment with anti-C5 therapies, a large proportion of people with PNH remain anemic, and some dependent on blood transfusions7,11–16. 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 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 period2,17. The primary endpoint is change from baseline Hb levels after 24 weeks of treatment with Fabhalta2,17. 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 randomization5,18. 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 pathway21,22. 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 condition25–31. 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 submissions32-35. Disclaimer This 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 nearly 300 million people worldwide. Reimagine medicine with us: Visit us at and connect with us on LinkedIn, Facebook, X/Twitter and Instagram. References Novartis. Data on file. 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: Accessed May, 2025. Montan I, Löwe B, Cella D, Mehnert A, Hinz A. General Population Norms for the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale. Value Health. 2018;21(11):1313-1321. doi:10.1016/ Cella D, Lai JS, Chang CH, Peterman A, Slavin M. Fatigue in cancer patients compared with fatigue in the general United States population. Cancer. 2002;94(2):528-538. doi:10.1002/cncr.10245 Peffault de Latour R, Röth A, Kulasekararaj AG, et al. Oral Iptacopan Monotherapy in Paroxysmal Nocturnal Hemoglobinuria. N Engl J Med. 2024;390(11):994-1008. doi:10.1056/NEJMoa2308695 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. 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. 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. 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. 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. 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 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. 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. NCT04558918. Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody Treatment (APPLY-PNH). Available from: Accessed May, 2025. Risitano AM, Han B, Ueda Y, et al. Oral Complement Factor B Inhibitor Iptacopan Monotherapy Improves Hemoglobin to Normal/Near-Normal Levels in Paroxysmal Nocturnal Hemoglobinuria Patients Naïve to Complement Inhibitors: Phase III APPOINT-PNH Trial. Presented at: 49th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT); April 23-36, 2023; Paris, France. NCT04820530. Study of Efficacy and Safety of Twice Daily Oral Iptacopan (LNP023) in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy (APPOINT-PNH). Available from: Accessed May, 2025. Kavanagh D, Bomback A, Vivarelli M, et al. Efficacy and Safety of Iptacopan in Patients with C3 Glomerulopathy: Results from the Phase 3 APPEAR-C3G Trial. Presented at European Renal Association (ERA) Congress; May 25, 2024; Stockholm, Sweden. Smith RJ, Kavanagh D, Vivarelli M, et al. Efficacy and safety of iptacopan in patients with C3 glomerulopathy: 12-Month results from the Phase 3 APPEAR-C3G study. Presented at American Society of Nephrology (ASN) Kidney Week 2024; October 23-27, 2024; San Diego, CA. Novartis. Press release. Novartis receives FDA approval for Fabhalta® (iptacopan), offering superior hemoglobin improvement in the absence of transfusions as the first oral monotherapy for adults with PNH. Available from: Accessed May, 2025. 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: Accessed May, 2025. Novartis. Press release. Novartis receives third FDA approval for oral Fabhalta® (iptacopan) – the first and only treatment approved in C3 glomerulopathy (C3G). Available from: Accessed May, 2025. Novartis. Press release. Novartis oral Fabhalta® (iptacopan) receives positive CHMP opinion for the treatment of adults living with C3 glomerulopathy (C3G). Available from: Accessed May, 2025. Fabhalta®. US FDA Prescribing information. East Hanover, NJ:Novartis Pharmaceuticals Corp; 2024. Available from: Accessed May, 2025. Fabhalta®. EMA Summary of Product Characteristics. Novartis Europharm Limited; 2024. Available from: Accessed May, 2025. Martín B, Smith RJH. C3 Glomerulopathy. In: Adam MP, Feldman J, Mirzaa GM, et al, eds. GeneReviews® [Internet]. Seattle, WA: University of Washington, Seattle; 1993-2025. Updated April 5, 2018. Available from: Accessed May, 2025. Schena FP, Esposito P, Rossini M. A Narrative Review on C3 Glomerulopathy: A Rare Renal Disease. Int J Mol Sci. 2020;21(2):525. Caravaca-Fontán F, Lucientes L, Cavero T, Praga M. Update on C3 Glomerulopathy: A Complement-Mediated Disease. Nephron. 2020;144(6):272-280. NCT04578834. Study of Efficacy and Safety of LNP023 in Primary IgA Nephropathy Patients (APPLAUSE-IgAN). Available from: Accessed May, 2025. NCT04889430. Efficacy and Safety of Iptacopan (LNP023) in Adult Patients With Atypical Hemolytic Uremic Syndrome Naive to Complement Inhibitor Therapy (APPELHUS). Available from: Accessed May, 2025. NCT05755386. Study of Efficacy and Safety of Iptacopan in Participants With IC-MPGN (APPARENT). Available from: Accessed May, 2025. NCT05268289. Study of Efficacy and Safety of LNP023 in Participants With Active Lupus Nephritis Class III-IV, +/- V. Available from: Accessed May, 2025. # # # Disclaimer: The above press release comes to you under an arrangement with GlobeNewswire. Business Upturn takes no editorial responsibility for the same. GlobeNewswire provides press release distribution services globally, with substantial operations in North America and Europe.
Yahoo
08-05-2025
- Health
- Yahoo
Emerging Treatments Drive Notable Changes in IgA Nephropathy Care Across Key Global Markets, According to Spherix Global Insights
Products such as Tarpeyo (Calliditas), Filspari (Travere) and now Novartis' Fabhalta and Venrafia provide physicians more options than ever before. EXTON, PA, May 08, 2025 (GLOBE NEWSWIRE) -- With multiple targeted therapies now available and SGLT2 inhibitors broadly embraced, nephrologists across the globe are redefining how they manage IgA Nephropathy (IgAN) patients. Data from Spherix Global Insights' comprehensive Patient Chart Dynamix™ services—spanning over 2,300 patient charts across the US, EU5, Japan, and China—demonstrate the magnitude of this shift, highlighting differences in treatment patterns, product adoption, and future intent across markets. As standard treatment practice increasingly incorporates SGLT2 inhibitors—initially reserved for diabetes and then diabetic kidney disease—nephrologists are initiating these agents earlier in the treatment algorithm, citing emerging data supporting their protective effects in CKD, including IgAN. Adoption has grown substantially year-over-year in all surveyed markets. While regional differences exist, the early use of SGLT2 inhibitors, in combination with RAASi, is now widely accepted as foundational care for non-dialysis IgAN patients. Overlaying this supportive care are newly launched agents designed specifically for IgAN. Calliditas' Tarpeyo® (budesonide) and Travere's Filspari® (sparsentan) have quickly gained traction in the US, with prescribing steadily moving upstream in the treatment paradigm. Indeed, nearly one in five US IgAN patients are currently managed with Tarpeyo, and Filspari's use has nearly doubled in the past year alone. Across Europe, Kinpeygo—Tarpeyo's EU label—has seen notable growth, especially in Germany and the UK. Filspari's limited availability at the time of fielding across many regions somewhat constrained its uptake, though future use intentions are high as access expands. In China, Nefecon (budesonide) has rapidly secured a foothold, with many patients already initiated on therapy. Despite different access landscapes, nephrologists are aligned on one key goal: reducing proteinuria to below 0.5g/day. Yet, many patients remain above this threshold despite therapy, underscoring the need for additional, more effective options. Enter Novartis, with Fabhalta® (iptacopan), recently approved in the US and garnering early interest for use in later lines of therapy. Vanrafia®, also from Novartis, has just received its first approval and is poised to further expand the treatment armamentarium. Together, these products—alongside pipeline agents targeting the complement system, APRIL/BAFF pathways, and other novel MOAs—represent a new chapter in IgAN care focused on underlying disease pathophysiology. Importantly, Spherix's data highlight regional nuances in how nephrologists view and sequence these therapies. In Japan, tonsillectomy remains a widely accepted option and is often paired with systemic corticosteroids. In contrast, physicians in the US and China are increasingly moving away from broad immunosuppression in favor of targeted mechanisms with improved safety profiles. While there is general agreement on first-line strategies, subsequent lines of therapy are in flux, shaped by clinical experience, product availability, and evolving guideline recommendations. Physicians continue to prioritize clinical data demonstrating both proteinuria reduction and eGFR preservation when evaluating new options, though safety warnings remain a barrier to adoption for some. Even so, enthusiasm for therapies with novel mechanisms continues to grow, particularly in markets where patients remain difficult to manage despite standard therapies. With over five years of continuous tracking, Spherix Global Insights has established the most robust and consistent global dataset on IgAN, offering the industry's most comprehensive view of how real-world treatment decisions have evolved and how emerging therapies are being integrated into clinical practice. By continuing to track real-world patient management trends and comparing data across markets, Spherix delivers actionable insights that support strategic decision-making for stakeholders across the nephrology landscape. Patient Chart Dynamix™ is an independent, data-driven service unveiling real patient management patterns through rigorous analysis of large-scale patient chart audits. Insights reveal the 'why' behind treatment decisions, include year over year trending to quantify key aspects of market evolution, and integrate specialists' attitudinal & demographic data to highlight differences between stated and actual treatment patterns. About Spherix Global Insights Spherix is a leading independent market intelligence and advisory firm that delivers commercial value to the global life sciences industry, across the brand lifecycle. The seasoned team of Spherix experts provides an unbiased and holistic view of the landscape within rapidly evolving specialty markets, including dermatology, gastroenterology, rheumatology, nephrology, neurology, ophthalmology, and hematology. Spherix clients stay ahead of the curve with the perspective of the extensive Spherix Physician Community. As a trusted advisor and industry thought leader, Spherix's unparalleled market insights and advisory services empower clients to make better decisions and unlock opportunities for growth. To learn more about Spherix Global Insights, visit or connect through LinkedIn. For more details on Spherix's primary market research reports and interactive dashboard offerings, visit or register here: Spherix Global Insights Contacts Tucker Hurtado, Nephrology Franchise Head NOTICE: All company, brand or product names in this press release are trademarks of their respective holders. The findings and opinions expressed within are based on Spherix Global Insight's analysis and do not imply a relationship with or endorsement of the companies or brands mentioned in this press release. CONTACT: Tucker Hurtado, Nephrology Franchise Head Spherix Global Insights 4848794284 in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
