CSL Vifor and Travere Therapeutics, Inc., (NASDAQ: TVTX) today announced that the European Commission has granted conditional marketing authorization (CMA) for FILSPARI (sparsentan) for the treatment of adults with primary IgAN with a urine protein excretion ≥1.0 g/day (or urine protein-to-creatinine ratio ≥0.75 g/g). The CMA is granted for all member states of the European Union, as well as in Iceland, Liechtenstein and Norway.

"This is a significant step forward for patients in Europe living with IgAN, a rare and serious condition, and a leading cause of end stage renal disease," said Prof. Dr. med. Jürgen Floege, Senior Professor, Div. Nephrology and Clinical Immunology at the University Hospital, RWTH Aachen, Germany, and steering committee member for the PROTECT clinical trial. "The approval of this innovative treatment is based on data from the only head-to-head phase-III clinical trial in IgAN. Adult patients with IgAN who are at high risk of progressing to kidney failure will now have access to a new therapy that significantly reduces proteinuria and slows the progression of kidney disease."

"The approval by the European Commission is an important milestone for the IgAN community in Europe and underscores our promise to develop and deliver innovative medicines in our areas of focus where there is unmet need," said Emmanuelle Lecomte Brisset, Senior Vice President and Head of Global Regulatory Affairs at CSL. "We look forward to working with our partners and EU member states to bring this innovative therapy to patients in Europe."

"As the first and only non-immunosuppressive therapy approved for IgAN, we believe FILSPARI offers clinicians the potential for a new foundational treatment for this rare kidney disease, replacing RAAS inhibition," said Eric Dube, Ph.D., President and Chief Executive Officer, Travere Therapeutics. "With this approval and the commercial strength and expertise of our partner, CSL Vifor, we look forward to people living with IgAN in Europe gaining access to this important medicine."  

The European Commission's decision follows the Committee for Medicinal Products for Human Use (CHMP)'s positive opinion in February 2024, based on results from the pivotal phase-III PROTECT study of FILSPARI in IgAN. 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.

CSL Vifor expects to launch FILSPARI in the first European markets in the second half of 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, www.cslvifor.com.

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 travere.com.

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.

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 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, in 404 patients ages 18 years and up with IgAN and persistent proteinuria despite receiving maximum tolerated dose and at least 50% of maximum­ labelled dose of ACE or ARB therapy. In August 2021, Travere announced the PROTECT Study met its primary endpoint at the pre-specified interim analysis . Based on the pre-specified, primary analyses set, after 36 weeks of treatment, patients receiving sparsentan achieved a mean reduction in proteinuria from baseline of 49.8%, compared to a mean reduction in proteinuria from baseline of 15.1% for irbesartan-treated patients (p<0.0001). The study's confirmatory secondary endpoint in the EU is eGFR chronic slope, measured from week 6 to week 110 of treatment, following the initial acute effect of randomized treatment. The confirmatory secondary endpoint in the U.S. is eGFR total slope from day 1 to week 110 of treatment. In September 2023, Travere announced topline two-year confirmatory secondary endpoint results from the PROTECT Study of sparsentan in IgAN. Sparsentan demonstrated long-term kidney function preservation and achieved a clinically meaningful difference in eGFR chronic and total slope versus irbesartan achieving statistical significance in eGFR chronic slope for purposes of regulatory review in the EU, and narrowly missing statistical significance in eGFR total slope. Patients who completed the PROTECT double-blind portion of the study on treatment were eligible to participate in the open-label extension of the trial. In PROTECT, the most common adverse reactions (≥ 5%) were peripheral edema, hypotension (including orthostatic hypotension), dizziness, hyperkalemia, and anemia.

About FILSPARI (sparsentan)

FILSPARI is a novel, 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). Pre-clinical data have shown that blockade of both endothelin type A and angiotensin II type 1 pathways in forms of rare chronic kidney disease, protects podocytes, prevents glomerulosclerosis and mesangial cell proliferation, and reduces proteinuria. 

For more information please refer to the product overview on the European Medicines Agency website.

FILSPARI was developed by Travere Therapeutics and has been granted Orphan Drug Designation for the treatment of IgAN in Europe and the U.S. FILSPARI is currently available in the U.S. and was granted accelerated approval by the US Food and Drug Administration in February 2023 based on reduction in proteinuria. CSL Vifor has been granted exclusive commercialization rights for FILSPARI in Europe, Australia and New Zealand.