England's NICE recommends FILSPARI® (sparsentan) as a treatment option for IgA nephropathy

Rhea-AI Summary

CSL Vifor announced that FILSPARI® (sparsentan) has received recommendation from England's National Institute for Health and Care Excellence (NICE) for treating IgA nephropathy. The treatment is approved for adults with urine protein excretion ≥1.0 g/day or urine protein-to-creatinine ratio ≥0.75 g/g. This marks the first non-immunosuppressive dual-action therapy recommended for this condition, which affects over 22,000 adults in England. The recommendation follows MHRA authorization in April 2025, with commercial availability expected from July 2025. IgA nephropathy, the most common primary glomerular disease worldwide, leads to kidney failure in 30-40% of patients within 10 years of diagnosis if not properly controlled. The NICE decision requires NHS England to fund the treatment within 90 days of final publication, expected on June 27, 2025.

Positive

• First non-immunosuppressive dual-action therapy approved for IgA nephropathy in England
• Large addressable market with over 22,000 adult patients in England
• Mandatory NHS funding within 90 days of final publication
• Commercial launch planned for H2 2025 with stock available from July 2025

Negative

• None.

Insights

Pharmaceutical Regulatory Expert

NICE recommendation of FILSPARI for IgA nephropathy represents significant market access achievement for CSL Vifor, expanding treatment options for this kidney disease.

The NICE recommendation of FILSPARI (sparsentan) represents a significant regulatory milestone for CSL Vifor in the UK market. This decision ensures the drug will be funded throughout England within 90 days of final publication (expected June 27, 2025), with commercial availability starting July 2025.

What makes this approval particularly notable is that FILSPARI is the first non-immunosuppressive dual-action therapy recommended for IgA nephropathy, a leading cause of kidney failure. The approval covers adults with substantial proteinuria (≥1.0 g/day or protein-to-creatinine ratio ≥0.75 g/g), though with continuation criteria based on treatment response.

IgA nephropathy affects approximately 22,000 adults in England alone, with 30-40% developing kidney failure within 10 years of diagnosis if not properly managed. The condition, while classified as rare, represents the most common primary glomerular disease globally.

The NICE recommendation follows the UK's MHRA authorization in April 2025, which was based on the Phase-III PROTECT trial results. This approval pathway demonstrates CSL Vifor's successful navigation of both clinical and health economic evaluations, as NICE's positive assessment indicates the therapy provides sufficient benefit and value for money to warrant routine use.

The expanded access in England complements CSL Vifor's strategic positioning in the rare kidney disease market, with commercial launch planned for the second half of 2025.

Nephrology Specialist

FILSPARI offers significant advancement for IgA nephropathy patients with its dual-action, non-immunosuppressive mechanism and proven proteinuria reduction.

FILSPARI (sparsentan) represents a transformative therapeutic option for IgA nephropathy management. Current treatment approaches have significant limitations, with many patients failing to achieve adequate proteinuria reduction despite maximal supportive care, leaving them at continued risk for disease progression.

The clinical significance of this approval stems from sparsentan's unique dual mechanism of action as a non-immunosuppressive therapy. Unlike conventional immunosuppressive treatments that broadly suppress immune function with associated side effects, sparsentan specifically targets the pathophysiological mechanisms driving IgA nephropathy progression.

Proteinuria reduction is the critical surrogate endpoint in IgA nephropathy management, as it directly correlates with kidney preservation. The Phase-III PROTECT trial that supported this approval demonstrated meaningful proteinuria reduction, addressing the central risk factor for disease progression.

The NICE recommendation specifically targets patients with significant proteinuria (≥1.0 g/day or protein-to-creatinine ratio ≥0.75 g/g), aligning with KDIGO guidelines identifying these patients as high-risk for progressive chronic kidney disease. The continuation criteria ensure the therapy is directed toward responsive patients, optimizing healthcare resource utilization.

For the typical IgA nephropathy patient—diagnosed around age 40 and facing potential kidney failure—this therapy addresses a critical therapeutic gap. With approximately 30-40% of patients developing kidney failure within a decade of diagnosis, FILSPARI provides a much-needed intervention that may delay or prevent progression to kidney failure, potentially reducing dialysis and transplantation needs in this population.

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 /PRNewswire/ -- 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, cslvifor.com.

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:

1. 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.
2. 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.
3. NICE Draft Final Guidance on sparsentan (May 2025).
4. 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.
5. Travere Therapeutics and CSL Vifor Announce Standard EU Approval of FILSPARI® (sparsentan) for IgA Nephropathy; press release (April 2025).
6. Pitcher D, et al. Long-Term Outcomes in IgA Nephropathy. Clin J Am Soc Nephrol. 2023;18(6):727–38.av.
7. Reich HN, et al. Remission of proteinuria improves prognosis in IgA nephropathy. J Am Soc Nephrol. 2007;18:3177–83.
8. Sharma S, et al. From Proteinuria to Fibrosis: An Update on Pathophysiology and Treatment Options. Kidney Blood Press Res. 2021;46:411−20.
9. 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/j.ekir.2021.02.018. PMID: 34169207; PMCID: PMC8207308.
10. European Medicines Agency (EMA). (2020) Orphan designation for the treatment of primary IgA nephropathy https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2345 (accessed May 2025).
11. 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.
12. KDIGO 2021 Clinical Practice Guideline for the Management of Glomerular Disease, Kidney International (2021) 100, S1-S276 https://kdigo.org/wp-content/uploads/2017/02/KDIGO-Glomerular-Diseases-Guideline-2021-English.pdf (accessed May 2025).
13. NKF Kidney Disease Stages https://kidneycareuk.org/kidney-disease-information/stages-of-kidney-disease/stages-of-chronic-kidney-disease-ckd/ (accessed May 2025).
14. NKF Haemodialysis https://kidneycareuk.org/kidney-disease-information/treatments/patient-info-haemodialysis-hd/ (accessed May 2025).
15. NKF Transplants for All https://www.kidney.org/transplantationNKUK Transplantation https://www.kidneyresearchuk.org/transplantation/ (accessed May 2025).
16. Mayo Clinic What is IgA Nephropathy? https://www.mayoclinic.org/diseases-conditions/iga-nephropathy/symptoms-causes/syc-20352268 (accessed May 2025).
17. 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.
18. Filspari EU131-SmPC_SPT_UK_200mg UK SmPC (May 2025).
19. Filspari EU131-SmPC_SPT_UK_400mg UK SmPC (May 2025).

CSL Vifor Media Contact

Thomas Hutter
+41 79 957 96 73
media@viforpharma.com

Job no: UK-SPT-25000110  Date: 23 May 2025

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SOURCE Vifor International AG (CSL Vifor)

FAQ

What is FILSPARI and what did NICE recommend it for?

FILSPARI (sparsentan) is a non-immunosuppressive dual-action therapy that NICE recommended for treating IgA nephropathy in adults with specific protein excretion levels. It's the first treatment of its kind recommended for this condition in England.

When will FILSPARI be available in the UK for IgA nephropathy patients?

CSL Vifor plans to launch FILSPARI in the UK in the second half of 2025, with commercial stock becoming available from July 2025.

How many people in England are affected by IgA nephropathy?

Over 22,000 adults in England are estimated to have IgA nephropathy, making it the most common type of primary glomerular disease.

What is the prognosis for untreated IgA nephropathy patients?

Without appropriate control, approximately 30-40% of IgA nephropathy patients develop kidney failure within 10 years of diagnosis.

What criteria did NICE set for FILSPARI treatment eligibility?

NICE recommended FILSPARI for adults with IgA nephropathy who have 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.