SAN DIEGO--(BUSINESS WIRE)---- $TVTX #FSGS--Travere Therapeutics, Inc., (Nasdaq: TVTX) today announced that the U.S. Food and Drug Administration (FDA) has informed the Company that following further review of the supplemental New Drug Application (sNDA) for FILSPARI® (sparsentan) in focal segmental glomerulosclerosis (FSGS), an advisory committee is no longer needed. The sNDA remains under review by the FDA with a Prescription Drug User Fee Act (PDUFA) target action date of January 13, 2026. "FSGS is a lea ...

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]