Latest news with #sparsentan
Medscape
28-05-2025
- Health
- Medscape
NICE Approves Sparsentan for Kidney Disease Treatment
The National Institute for Health and Care Excellence has recommended sparsentan (Filspari, Vifor Pharma) for treating primary immunoglobulin A nephropathy in adults. The recommendation reverses NICE's February decision to reject NHS funding for the drug. The regulator had previously said available evidence did not demonstrate value for money. The manufacturer provided additional analyses and agreed to a price discount, leading to the positive recommendation. Clinical Benefits Demonstrated Sparsentan reduces urine protein-to-creatinine ratio (UPCR) more effectively than standard treatment. The drug may also better maintain kidney function, NICE said. In its final draft guidance, the regulator recommended sparsentan for IgAN patients with a urine protein excretion of 1.0 g/day or more, or a UPCR of 0.75 g/g or higher. Treatment should cease after 36 weeks if a patient's UPCR remains at or above 1.76 g/g and has not reduced by at least 20%. Significant Patient Impact IgAN affects more than 18,000 people in England. It ranks among the most common causes of chronic kidney disease and kidney failure. Over 4200 people with chronic kidney disease could benefit from NICE's recommendation. Standard care for IgAN includes angiotensin-2 receptor blockers such as irbesartan. Mechanism of Action Sparsentan works by blocking the receptors for two hormones —endothelin-1 and angiotensin II — that cause kidney damage. By blocking these receptors, the drug reduces the amount of proteinuria and slows down the progression of kidney damage. Clinical trial evidence has shown that sparsentan reduces UPCR more than irbesartan. Evidence also suggests that sparsentan is better at maintaining kidney function than irbesartan, but this was uncertain, NICE explained. PROTECT Study Results The regulator's recommendation was informed by the outcome of the PROTECT study. The double-blind, randomised, active-controlled, phase 3 trial included patients aged 18 years or older with biopsy-proven primary IgAN and proteinuria of at least 1.0 g daily despite maximised renin-angiotensin system inhibition for a minimum of 12 weeks. Patients were randomly assigned to either receive 400 mg of oral sparsentan or 300 mg of oral irbesartan, both taken once daily. The sparsentan group showed slower rates of estimated glomerular filtration rate (eGFR) decline. At 36 weeks, sparsentan had significantly reduced proteinuria, a reduction that continued throughout the study. At 110 weeks, proteinuria, as determined by the change from baseline in UPCR, was 40% lower in the sparsentan group compared with irbesartan patients. The composite kidney failure endpoint was reached by 9% of patients in the sparsentan group compared with 13% of those in the irbesartan group. The researchers concluded that treatment with sparsentan versus maximally titrated irbesartan in patients with IgAN resulted in significant reductions in proteinuria and preservation of kidney function. Treatment Advantages Sparsentan is taken as a once-daily tablet for long-term use. This differs from targeted-release budesonide, which is limited to 9 months' duration. NICE said it expected the treatment to reduce NHS pressure by preventing or delaying progression to end-stage renal disease requiring dialysis or transplant. Helen Knight, director of medicines evaluation at NICE, highlighted the limited treatment options available for the disease. She said that sparsentan offered long-term benefits to patients and 'could make a huge difference to people's lives by delaying kidney failure'. Fiona Loud, policy director at Kidney Care UK, welcomed the guidance. She noted that IgAN typically affects younger patients, disrupting lives when people have young families or are starting careers. 'We're pleased that this new treatment for IgAN will now be an option for patients who need it,' Loud said.
Yahoo
26-05-2025
- Business
- Yahoo
CSL (ASX:CSL) Secures NICE Approval For Sparsentan Use In NHS England
CSL saw its stock price rise by 3% over the last month, amid important developments and a fluctuating market environment. CSL Vifor's notable progress with the National Institute for Health and Care Excellence recommending sparsentan for primary IgA nephropathy treatment may have provided positive momentum. This significant medical endorsement, alongside CSL's continued engagement with shareholders through meetings, could have added weight to its share performance. Meanwhile, broader market volatility, influenced by global trade tensions and declining major indexes, may have tempered the extent of CSL's gains, which align closely with general market movement, as the market faced a 1% decline. We've spotted 1 possible red flag for CSL you should be aware of. The latest GPUs need a type of rare earth metal called Dysprosium and there are only 24 companies in the world exploring or producing it. Find the list for free. The recent developments regarding CSL's approval for sparsentan, a treatment recommended by the National Institute for Health and Care Excellence, may enhance the company's market position, potentially fueling revenue growth. Over the past three years, however, CSL's total return, inclusive of share price changes and dividends, recorded a decline of 4.64%. This contrasts against a broader 1-year underperformance both relative to the Australian market's 4.9% increase and the Australian Biotechs industry's -9.4% decrease, highlighting a need to bridge the performance gap with new advancements. Looking forward, the impact of these medical endorsements might influence CSL's revenue and earnings forecasts positively, with anticipated expansion in the HAE and vaccine sectors playing a crucial role. Despite the current share price of A$251.13 being lower than the consensus analyst price target of A$311.94, suggesting a potential upswing, the market's fluctuations must be considered in investment evaluations. As CSL continues to hone operational efficiencies and product rollouts, assessing these factors against earnings projections will be vital in understanding its long-term trajectory. According our valuation report, there's an indication that CSL's share price might be on the cheaper side. This article by Simply Wall St is general in nature. We provide commentary based on historical data and analyst forecasts only using an unbiased methodology and our articles are not intended to be financial advice. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. We aim to bring you long-term focused analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Simply Wall St has no position in any stocks mentioned. Companies discussed in this article include ASX:CSL. This article was originally published by Simply Wall St. Have feedback on this article? Concerned about the content? with us directly. Alternatively, email editorial-team@
Yahoo
23-05-2025
- Health
- Yahoo
England's NICE recommends FILSPARI® (sparsentan) as a treatment option for IgA nephropathy
First non-immunosuppressive dual-action therapy recommended by NICE for eligible patients with IgA nephropathy, a leading cause of kidney failure 1-3 NICE's recommendation is based on clinically meaningful results from the phase-III PROTECT trial 4 ST. GALLEN, Switzerland, May 23, 2025 /CNW/ -- CSL Vifor is pleased to announce that the National Institute for Health and Care Excellence (NICE) has published final draft guidance recommending that sparsentan can be used in the NHS in England as an option to treat primary IgA nephropathy in adults with a urine protein excretion of 1.0 g/day or more, or a urine protein-to-creatinine ratio of 0.75 g/g or more.3 NICE has provided guidance to ensure that only patients responding to treatment continue.3 The decision follows authorisation from the UK's Medicines and Healthcare products Regulatory Agency (MHRA) in April 2025.5 What this means in practice is that there is enough evidence to show that sparsentan provides benefits and value for money, so it can be used routinely if it is considered the most suitable treatment option in this population.3 Sparsentan must be funded in England within 90 days of final publication of this guidance3 which is expected to be 27 June 2025. Professor Jonathan Barratt, Professor of Renal Medicine at University Leicester, UK, welcomed the NICE decision as a major advancement in the treatment of IgA nephropathy in the UK. "IgA nephropathy is a condition with an average age at diagnosis of around 40 years.6 Due to disease progression, a patient's kidneys may fail. Treatments, such as sparsentan, that have been developed for IgA nephropathy are urgently needed, our goal being to improve outcomes for these patients." IgA nephropathy is characterised by the buildup of a faulty version of immunoglobulin A (IgA), which accumulates in clusters in small blood vessels in the kidney, called glomeruli, that filter the blood. These clumps damage the glomeruli causing leakage of blood (haematuria) and protein (proteinuria) into the urine resulting in a progressive loss of kidney function. Proteinuria is a major risk factor for IgA nephropathy progression, increasing the risk of kidney failure.6-8 Despite current treatments, some patients with IgA nephropathy do not achieve adequate proteinuria reduction and remain at risk of progression.9 Although classified as rare, IgA nephropathy is the most common type of primary glomerular disease worldwide, with over 22,000 adults estimated to have the condition in England.10 Patients generally face a poor prognosis if the condition is not appropriately controlled, with approximately 30-40% of patients developing kidney failure within 10 years of diagnosis.11 Current medical treatment guidelines by KDIGO (Kidney Disease, Improving Global Outcomes) state that patients who are at high risk of progressive chronic kidney disease, despite maximal supportive care, are those with persistent urine protein excretion >1 g/day.12 Underscoring the importance of the NICE recommendation for IgA nephropathy patients and their communities, Dr. Vinicius Gomes De Lima, Head of Global Medical Affairs at CSL Vifor said: "We are very pleased that NICE recognised the value of our innovative therapy which helps to address a clear unmet medical need in patients with IgA nephropathy. We look forward to working with the National Health Service to ensure access to this important medicine as soon as possible as we continue to deliver on our promise to patients." CSL Vifor expects to launch sparsentan in the UK in the second half of 2025; commercial stock will be available from July 2025. Notes to Editors On 15th April 2025, the MHRA granted the marketing authorisation for sparsentan based on the final results of the Phase 3 PROTECT double blind study.5 About CSL Vifor CSL Vifor is a global partner of choice for pharmaceuticals and innovative, leading therapies in iron deficiency and nephrology. We specialise in strategic global partnering, in-licensing and developing, manufacturing and marketing pharmaceutical products for precision healthcare, aiming to help patients around the world lead better, healthier lives. Headquartered in St. Gallen, Switzerland, CSL Vifor also includes the joint company Vifor Fresenius Medical Care Renal Pharma (with Fresenius Medical Care). The parent company, CSL (ASX: CSL; USOTC: CSLLY), headquartered in Melbourne, Australia, employs 32,000 people and delivers its lifesaving therapies to people in more than 100 countries. For more information about CSL Vifor visit, About IgA nephropathy Primary immunoglobulin A nephropathy (IgA nephropathy) is a rare, progressive type of chronic kidney disease (CKD) that is often diagnosed in adults before the age of 40 years.6 CKD is characterised by abnormalities of kidney function or structure that have been present for more than three months and can be categorised into five stages dependent on functionality of the kidney.13 Dialysis (a medical treatment used to artificially filter waste products and excess fluids from the blood when the kidneys are unable to perform this function adequately)14 or kidney transplantation is recommended for patients whose kidneys have reached an advanced stage (typically, stage 5).15 More than 60 per cent of adult patients diagnosed with IgA nephropathy are in CKD stage 3 or higher.6 Patients with this condition may experience blood in the urine (red or dark brown urine), foamy urine from protein leaking into the urine, flank pain, swelling (oedema), high blood pressure, and fatigue.16 About FILSPARI® (sparsentan) Sparsentan was developed by Travere Therapeutics and has been granted Orphan Drug Designation for the treatment of IgA nephropathy in the UK, Europe and the U.S. Sparsentan is currently available in the U.S. and first markets in Europe. CSL Vifor has been granted exclusive commercialisation rights for sparsentan in Europe, Australia and New Zealand. Sparsentan is anticipated to be available to patients in the UK in the second half of 2025. Sparsentan is the first and only non-immunosuppressive treatment for IgA nephropathy that has two modes of action.1 This single molecule functions as a high affinity, dual-acting antagonist of both the endothelin A receptor (ETAR) and the angiotensin II subtype 1 receptor (AT1R).4 Sparsentan inhibits activation of both ETAR and AT1R, both of which play a role in regulating processes in the kidney, such as inflammation, that lead to progression of kidney damage.4 About PROTECT NICE's recommendation is based on data from the pivotal Phase 3 PROTECT study4 of sparsentan in IgA nephropathy, one of the largest interventional studies to date in IgA nephropathy and the only head-to-head trial in this rare kidney disease. It is a global, randomised, multicentre, double-blind, parallel-arm, active-controlled clinical trial evaluating the safety and efficacy of 400 mg of sparsentan, compared to 300 mg of irbesartan (an angiotensin II receptor blocker(ARB)), in 404 patients ages 18 years and up with IgA nephropathy and persistent proteinuria despite receiving at least 50% of maximum label dose and maximally tolerated angiotensin-converting enzyme (ACE) inhibitors or ARB therapy.4,17 The PROTECT study met its primary endpoint at the pre-specified interim analysis with statistical significance.4 After 36 weeks of treatment, patients receiving sparsentan (n=202) achieved a mean reduction in proteinuria from baseline of 49.8 percent, compared to a mean reduction in proteinuria from baseline of 15.1 percent for irbesartan-treated patients (n=202).4,17 Treatment emergent adverse events and serious adverse events were well-balanced between sparsentan and irbesartan, except for dizziness (30 [15%] vs 13 [6%] patients) and hypotension (26 (13%] vs eight (4%] patients).4 For more information, please refer to the Summary of Product Characteristics (SmPC).18,19 References: Trachtman H, et al. Sparsentan: the first and only non-immunosuppressive therapy for the reduction of proteinuria in IgA nephropathy. Expert Rev Clin Immunol. 2024 Jun;20(6):571-576. doi: 10.1080/1744666X.2024.2319132. Komers R, et al. Dual inhibition of renin-angiotensin-aldosterone system and endothelin-1 in treatment of chronic kidney disease. Am J Physiol Regul Integr Comp Physiol. 2016 May 15;310(10):R877-84. doi: 10.1152/ajpregu.00425.2015. NICE Draft Final Guidance on sparsentan (May 2025). Rovin BH, et al. Efficacy and safety of sparsentan versus irbesartan in patients with IgA nephropathy (PROTECT): 2-year results from a randomised, active-controlled, phase 3 trial. Lancet. 2023 Dec 2;402(10417):2077-2090. doi: 10.1016/S0140-6736(23)02302-4. Travere Therapeutics and CSL Vifor Announce Standard EU Approval of FILSPARI® (sparsentan) for IgA Nephropathy; press release (April 2025). Pitcher D, et al. Long-Term Outcomes in IgA Nephropathy. Clin J Am Soc Nephrol. 2023;18(6):727– Reich HN, et al. Remission of proteinuria improves prognosis in IgA nephropathy. J Am Soc Nephrol. 2007;18:3177–83. Sharma S, et al. From Proteinuria to Fibrosis: An Update on Pathophysiology and Treatment Options. Kidney Blood Press Res. 2021;46:411−20. Bagchi S, et al. Supportive Management of IgA Nephropathy With Renin-Angiotensin Blockade, the AIIMS Primary IgA Nephropathy Cohort (APPROACH) Study. Kidney Int Rep. 2021 Feb 26;6(6):1661-1668. doi: 10.1016/ PMID: 34169207; PMCID: PMC8207308. European Medicines Agency (EMA). (2020) Orphan designation for the treatment of primary IgA nephropathy (accessed May 2025). Barratt J, et al. Therapy of IgA nephropathy: time for a paradigm change. Front Med (Lausanne). 2024 Aug 15;11:1461879. doi: 10.3389/fmed.2024.1461879. PMID: 39211339; PMCID: PMC11358106. KDIGO 2021 Clinical Practice Guideline for the Management of Glomerular Disease, Kidney International (2021) 100, S1-S276 (accessed May 2025). NKF Kidney Disease Stages (accessed May 2025). NKF Haemodialysis (accessed May 2025). NKF Transplants for All Transplantation (accessed May 2025). Mayo Clinic What is IgA Nephropathy? (accessed May 2025). Heerspink HJL, et al. PROTECT Investigators. Sparsentan in patients with IgA nephropathy: a prespecified interim analysis from a randomised, double-blind, active-controlled clinical trial. Lancet. 2023 May 13;401(10388):1584-1594. doi: 10.1016/S0140-6736(23)00569-X. Epub 2023 Apr 1. PMID: 37015244. Filspari EU131-SmPC_SPT_UK_200mg UK SmPC (May 2025). Filspari EU131-SmPC_SPT_UK_400mg UK SmPC (May 2025). CSL Vifor Media Contact Thomas Hutter+41 79 957 96 73media@ Job no: UK-SPT-25000110 Date: 23 May 2025 View original content to download multimedia: SOURCE Vifor International AG (CSL Vifor) View original content to download multimedia: 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
Cision Canada
29-04-2025
- Business
- Cision Canada
CSL Vifor and Travere Therapeutics announce standard EU approval for FILSPARI® in IgA Nephropathy
European Commission converts conditional approval of FILSPARI (sparsentan) into standard marketing authorization for the treatment of IgA Nephropathy (IgAN) Decision follows positive recommendation from Committee for Medicinal Products for Human Use (CHMP) from February 2025 EU approval is based on the complete data set from the phase-III PROTECT study ST. GALLEN, Switzerland and SAN DIEGO, April 29, 2025 /CNW/ -- CSL Vifor and Travere Therapeutics, Inc., (NASDAQ: TVTX) are pleased to announce that the European Commission has approved the conversion of the conditional marketing approval (CMA) into a standard marketing authorization (MA) for FILSPARI for the treatment of adults with primary IgA nephropathy with a urine protein excretion ≥1.0 g/day (or urine protein-to-creatinine ratio ≥0.75 g/g). Standard MA is granted for all member states of the European Union, as well as in Iceland, Liechtenstein and Norway. "The decision by the European Commission is an important advancement for people living with IgAN in the EU", said Dr. Vinicius Gomes De Lima, Head of Global Medical Affairs at CSL Vifor. "The standard approval, granted without changes to the indication, underscores the value of our clinical data, the dedication of our teams, and our ongoing commitment to deliver on our promise for patients. We look forward to continuing working closely with healthcare professionals, patient communities, and regulatory bodies to ensure access to FILSPARI across Europe." The European Commission's standard approval of FILSPARI is a meaningful step forward for people living with IgA nephropathy across Europe," said Dr. Jula Inrig, Chief Medical Officer at Travere Therapeutics. "This decision not only validates the strength of the phase-III PROTECT study results but also reinforces our deep commitment to this rare kidney disease community. We remain dedicated to working with our partners, regulators, and healthcare providers to expand access and improve outcomes for those affected by IgAN." The European Commission's decision follows CHMP's recommendation to convert the CMA to standard MA from February 2025. The approval is based on a comprehensive clinical data set, including positive confirmatory results from the pivotal phase-III PROTECT study demonstrating that FILSPARI significantly slowed kidney function decline over two years compared to irbesartan. FILSPARI is the only Dual Endothelin Angiotensin Receptor Antagonist (DEARA), a non-immunosuppressive therapy for the treatment of IgAN approved in Europe and is currently available in Germany, Austria and Switzerland, following the European Commission's conditional marketing authorization in April 2024 About CSL Vifor CSL Vifor is a global partner of choice for pharmaceuticals and innovative, leading therapies in iron deficiency and nephrology. We specialize in strategic global partnering, in-licensing and developing, manufacturing and marketing pharmaceutical products for precision healthcare, aiming to help patients around the world lead better, healthier lives. Headquartered in St. Gallen, Switzerland, CSL Vifor also includes the joint company Vifor Fresenius Medical Care Renal Pharma (with Fresenius Medical Care). The parent company, CSL (ASX: CSL; USOTC: CSLLY), headquartered in Melbourne, Australia, employs 32,000 people and delivers its lifesaving therapies to people in more than 100 countries. For more information about CSL Vifor visit, About Travere Therapeutics At Travere Therapeutics, we are in rare for life. We are a biopharmaceutical company that comes together every day to help patients, families and caregivers of all backgrounds as they navigate life with a rare disease. On this path, we know the need for treatment options is urgent – that is why our global team works with the rare disease community to identify, develop and deliver life-changing therapies. In pursuit of this mission, we continuously seek to understand the diverse perspectives of rare patients and to courageously forge new paths to make a difference in their lives and provide hope – today and tomorrow. For more information, visit About IgA Nephropathy (IgAN) IgAN, also called Berger's disease, is a rare progressive kidney disease characterized by the buildup of immunoglobulin A (IgA), a protein that helps the body fight infections, in the kidneys. The deposits of IgA cause a breakdown of the normal filtering mechanisms in the kidney, leading to blood in the urine (hematuria), protein in the urine (proteinuria) and a progressive loss of kidney function. Other symptoms of IgAN may include swelling (edema) and high blood pressure. While rare, IgAN is the most common type of primary glomerular disease worldwide and a leading cause of kidney failure. IgAN is estimated to affect up to 250,000 people in the licensed territories (Europe, Australia and New Zealand). About the PROTECT study The PROTECT Study is one of the largest interventional studies to date in IgA nephropathy (IgAN) and the only head-to-head vs. comparator trial in this rare kidney disease. It is a global, randomized, multicenter, double-blind, parallel-arm, active-controlled clinical trial evaluating the safety and efficacy of 400 mg of sparsentan, compared to 300 mg of irbesartan (an angiotensin II receptor blocker(ARB)), in 404 patients ages 18 years and up with IgA nephropathy and persistent proteinuria despite receiving at least 50% of maximum label dose and maximally tolerated angiotensin-converting enzyme (ACE) inhibitors or ARB therapy. The PROTECT study met its primary endpoint at the pre-specified interim analysis with statistical significance. After 36 weeks of treatment, patients receiving FILSPARI achieved a mean reduction in proteinuria from baseline of 49.8 percent, compared to a mean reduction in proteinuria from baseline of 15.1 percent for irbesartan-treated patients. The two-year confirmatory results from the study showed treatment with FILSPARI achieved statistical significance on the eGFR chronic slope endpoint versus irbesartan and demonstrated clinically meaningful kidney function preservation. eGFR is a blood test that measure how well kidneys filter waste products from blood. Treatment emergent adverse events were well-balanced between sparsentan and irbesartan, except for dizziness and hypotension. About FILSPARI (sparsentan) FILSPARI is an innovative, non-immunosuppressive, single-molecule, dual endothelin angiotensin receptor antagonist with high selectivity for the endothelin A receptor (ETAR) and the angiotensin II subtype 1 receptor (AT1R). FILSPARI was developed by Travere Therapeutics and has been granted Orphan Drug Designation for the treatment of IgA nephropathy in the UK, Europe and the U.S. FILSPARI is currently available in the U.S. and first markets in Europe. CSL Vifor has been granted exclusive commercialization rights for FILSPARI in Europe, Australia and New Zealand. For more information, please refer to the Summary of Product Characteristics (SmPC). CSL Vifor Media Contact Travere Therapeutics: Investors 888-969-7879 [email protected] Media 888-969-7879 [email protected] SOURCE Vifor International AG (CSL Vifor)
