CSL Vifor and Travere Therapeutics announce EMA has accepted for review the Conditional Marketing Authorization application for sparsentan for the treatment of IgA Nephropathy

A review decision by the European Medicines Agency (EMA) is expected in the second half of 2023

If approved, sparsentan will be a first-in-class treatment to address the significant unmet medical need in IgA nephropathy (IgAN) in Europe

ST. GALLEN, Switzerland & SAN DIEGO, CA, USA I August 22, 2022 I CSL Vifor and Travere Therapeutics, Inc. (NASDAQ: TVTX) today announced that the EMA has accepted for review the Conditional Marketing Authorization (CMA) application for sparsentan for the treatment of IgAN, a rare kidney disorder and a leading cause of end-stage kidney disease (ESKD). The EMA will review the application under the centralized marketing authorization procedure and a review decision on a potential approval is expected in the second half of 2023.

“The acceptance of the EU regulatory application for sparsentan marks a major milestone towards our goal of bringing this first-in-class therapy to the patients suffering from IgAN, for which there are currently no approved non-immunosuppresive therapies,” commented Klaus Henning Jensen, Chief Medical Officer of CSL Vifor. “We look forward to working closely with our partner, Travere, through the EMA review process with the aim to bring this innovative treatment option to patients living with IgAN in Europe.”

“Following U.S. FDA’s acceptance and granting of priority review of the NDA for sparsentan for IgA nephropathy in the U.S., we continue to make great progress towards our goal of enabling sparsentan to become a new treatment standard for rare kidney disorders, if approved,” said Jula Inrig, M.D. chief medical officer of Travere Therapeutics. “The acceptance of the CMA application marks an important next step on our pathway to expanding access to sparsentan as the first non-immunosuppressive treatment option for IgA nephropathy in Europe, if approved. We look forward to continuing to collaborate with our partners at CSL Vifor and with the EMA throughout the review process.”

The EMA filing is supported by positive topline interim results from the ongoing pivotal phase-III PROTECT Study of sparsentan in IgAN, as well as supportive data from additional clinical studies. The PROTECT Study met its pre-specified interim primary efficacy endpoint with statistical significance. After 36 weeks of treatment, patients receiving sparsentan 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. Preliminary results at the time of the interim assessment suggested that sparsentan had been generally well-tolerated to date in the study and consistent with its overall observed safety profile.

If approved, sparsentan would receive CMA in all member states of the European Union, as well as in Iceland, Liechtenstein and Norway.

Sparsentan is currently also being evaluated in the pivotal phase-III DUPLEX Study for the treatment of focal segmental glomerulosclerosis (FSGS), another rare progressive kidney disorder and leading cause of end-stage kidney disease. Sparsentan has been granted Orphan Drug Designation for the treatment of IgAN and FSGS in Europe and in the U.S.

About CSL Vifor

CSL Vifor is a global partner of choice for pharmaceuticals and innovative, leading therapies in iron deficiency, dialysis and nephrology & rare disease. 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 30,000 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

IgA nephropathy (IgAN), also called Berger's disease, is a rare progressive kidney disorder 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 glomerulonephritis worldwide and a leading cause of end-stage kidney disease (ESKD). IgAN is estimated to affect more than 100,000 people in the U.S. and is one of the most common glomerular diseases in Europe and Japan. There are currently no approved non-immunosuppressive treatments indicated for IgAN.

About the PROTECT study

The ongoing PROTECT Study is one of the largest interventional studies to date in IgAN. It is a global, randomized, multicenter, double-blind, parallel-arm, active-controlled clinical trial evaluating the safety and efficacy of 400mg of sparsentan, compared to 300mg of irbesartan, in 404 patients ages 18 years and up with IgAN and persistent proteinuria despite available ACE or ARB therapy. In August 2021, the Company announced the PROTECT Study met its pre-specified interim primary efficacy endpoint with statistical significance. After 36 weeks of treatment, patients receiving sparsentan 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 (p<0.0001). The Company believes that preliminary eGFR data available at the time of the interim analysis are indicative of a potential clinically meaningful treatment effect after two years of treatment. Preliminary results at the time of the interim assessment suggested that sparsentan had been generally well-tolerated to date in the study and consistent with its overall observed safety profile. The PROTECT Study is fully enrolled and is scheduled to continue as planned on a blinded basis to assess the treatment effect on eGFR slope over 110 weeks in the confirmatory endpoint analysis. Topline results from the confirmatory endpoint analysis are expected in the second half of 2023.

About sparsentan

Sparsentan, a Dual Endothelin Angiotensin Receptor Antagonist (DEARA), is a novel investigational product candidate selectively targeting 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.

Sparsentan is also currently being evaluated in the pivotal phase-III DUPLEX study for the treatment of FSGS. In February 2021, Travere announced that the ongoing DUPLEX study of sparsentan in FSGS achieved its pre-specified interim FSGS partial remission of proteinuria endpoint (FPRE) with statistical significance. FPRE is a clinically meaningful endpoint defined as urine protein-to-creatinine ratio (UP/C) ≤1.5 g/g and a >40 percent reduction in UP/C from baseline. After 36 weeks of treatment, 42.0 percent of patients receiving sparsentan achieved FPRE, compared to 26.0 percent of irbesartan-treated patients. Preliminary results from the interim analysis suggest that at the time of the interim assessment, sparsentan had been generally well-tolerated and shown a comparable safety profile to irbesartan. In the phase-II DUET study of sparsentan in FSGS, the combined treatment group met its primary efficacy endpoint, demonstrating a greater than two-fold reduction in proteinuria compared to irbesartan, and was generally well tolerated after the eight-week, double-blind treatment period. Irbesartan is part of a class of drugs used to manage FSGS and IgAN. If approved for both indications, sparsentan could potentially be the first medicine approved for both FSGS and IgAN.

SOURCE: CSL Vifor

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