Core Insights - Travere Therapeutics, Inc. will present 11 abstracts at the American Society of Nephrology Kidney Week 2025, including a late-breaking poster [1] Company Developments - The company will showcase a late-breaking analysis from the DUPLEX Study focused on focal segmental glomerulosclerosis (FSGS) [1] - The analysis indicates that patients treated with FILSPARI® (sparsentan) were more likely to achieve proteinuria levels below 0 [1]

Core Insights - The updated KDIGO 2025 clinical practice guidelines for IgA Nephropathy emphasize the importance of achieving remission of proteinuria and slowing eGFR decline as key treatment goals [2][4] - FILSPARI (sparsentan) is highlighted as a first-line treatment option for IgA Nephropathy, demonstrating superior efficacy compared to traditional RASi therapies [3][4] Treatment Goals - The guidelines define remission of proteinuria as less than 0.5 g/day, ideally at less than 0.3 g/day, and emphasize the need to slow the decline of eGFR [2] - A treatment approach targeting both IgAN-induced nephron loss and IgA formation is recommended to meet these goals [2] FILSPARI's Role - FILSPARI is recognized as the only Dual Endothelin Angiotensin Receptor Antagonist (DEARA) and has shown proven efficacy in clinical trials, with more patients enrolled in the PROTECT study than in all prior RASi trials combined [3][4] - The PROTECT study demonstrated that patients receiving FILSPARI achieved a mean reduction in proteinuria of 49.8%, significantly higher than the 15.1% reduction seen in irbesartan-treated patients [7] Clinical Significance - The updated guidelines provide clarity for clinicians and patients, aiming to improve long-term kidney outcomes for those with IgA Nephropathy [4] - The inclusion of FILSPARI in the guidelines reflects its favorable benefit-risk profile and its role in reducing proteinuria and slowing kidney function loss [4][6] Company Background - CSL Vifor specializes in pharmaceuticals for iron deficiency and nephrology, focusing on strategic global partnerships and innovative therapies [8] - Travere Therapeutics is dedicated to developing treatments for rare diseases, emphasizing the urgent need for effective options in the IgA Nephropathy community [10]

Core Viewpoint - Travere Therapeutics, Inc. announced that the FDA has informed the company that an advisory committee is no longer needed for the supplemental New Drug Application (sNDA) for FILSPARI® (sparsentan) in focal segmental glomerulosclerosis (FSGS) [1] Group 1 - The sNDA for FILSPARI® remains under review by the FDA [1] - The Prescription Drug User Fee Act (PDUFA) target action date is set for January 13, 2026 [1]

Core Insights - NICE has recommended sparsentan as the first non-immunosuppressive dual-action therapy for primary IgA nephropathy in eligible patients, marking a significant advancement in treatment options [1][2][3] - The recommendation is based on the positive results from the Phase 3 PROTECT trial, which demonstrated sparsentan's efficacy in reducing proteinuria compared to irbesartan [1][8] Company Overview - CSL Vifor is a global partner specializing in pharmaceuticals and innovative therapies, particularly in iron deficiency and nephrology, with a focus on strategic global partnerships and precision healthcare [3][4] - The company plans to launch sparsentan in the UK in the second half of 2025, with commercial stock expected to be available from July 2025 [2][6] Industry Context - IgA nephropathy is the most common type of primary glomerular disease worldwide, affecting over 22,000 adults in England, with a significant risk of kidney failure if not adequately treated [2][5] - Current treatment guidelines indicate that patients with persistent urine protein excretion greater than 1 g/day are at high risk for progressive chronic kidney disease, highlighting the need for effective therapies like sparsentan [2][5][8] Clinical Trial Insights - The PROTECT trial involved 404 patients and showed that sparsentan achieved a mean reduction in proteinuria of 49.8% after 36 weeks, compared to 15.1% for irbesartan [8] - The trial was notable for being one of the largest interventional studies in IgA nephropathy and the only head-to-head trial in this area [8] Regulatory Milestones - The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) granted marketing authorization for sparsentan in April 2025, paving the way for its use in the NHS [1][3] - NICE's guidance mandates that sparsentan must be funded within 90 days of the final publication, expected on June 27, 2025 [1][2]

Core Viewpoint - Travere Therapeutics has submitted a supplemental New Drug Application (sNDA) to the FDA for FILSPARI, aiming for it to be the first FDA-approved treatment for focal segmental glomerulosclerosis (FSGS), a rare kidney condition [1][2] Company Overview - Travere Therapeutics is focused on developing therapies for rare diseases, emphasizing the urgent need for effective treatment options for conditions like FSGS [2][8] - FILSPARI is currently approved for slowing kidney function decline in adults with IgA nephropathy and is a non-immunosuppressive oral medication [2][9] Clinical Studies - The sNDA submission is supported by results from the Phase 3 DUPLEX Study and the Phase 2 DUET Study, which are among the largest interventional studies in FSGS [1][6] - The DUPLEX Study achieved its interim endpoint with statistical significance at 36 weeks, showing significant proteinuria reduction and a lower rate of end-stage kidney disease compared to the active control [6][7] - The DUET Study demonstrated a greater than two-fold reduction in proteinuria compared to irbesartan, with a consistent safety profile across trials [7] Regulatory Process - The FDA has 60 days to determine whether to accept the sNDA for review, with an expected notice in the second quarter of 2025 [3] - The FDA has indicated that REMS monitoring for embryo-fetal toxicity is no longer necessary, and the company plans to submit a modification to the REMS [4] Disease Context - FSGS is a rare kidney disorder affecting over 40,000 patients in the U.S., characterized by progressive scarring of the kidney and leading to kidney failure [5] - There are currently no FDA-approved pharmacologic therapies for FSGS, highlighting the significance of FILSPARI's potential approval [5]