CSL Vifor and Travere Therapeutics announce sparsentan receives positive CHMP opinion for the treatment of IgA nephropathy

Rhea-AI Summary

The CHMP recommends conditional marketing authorization for sparsentan in Europe for the treatment of IgA nephropathy, a rare kidney disorder. If approved, sparsentan will be the first non-immunosuppressive, single-molecule, dual endothelin angiotensin receptor antagonist for IgAN patients. The decision is expected in Q2 2024.

Medical Research Analyst

The recommendation by the CHMP for the approval of sparsentan represents a significant milestone for both Travere Therapeutics and the medical community involved in treating IgA nephropathy (IgAN). The potential market authorization in Europe could open a new revenue stream for Travere Therapeutics, considering the current lack of non-immunosuppressive treatments for IgAN in the region. The dual endothelin angiotensin receptor antagonist mechanism of sparsentan offers a novel approach that, as suggested by the PROTECT study, may reduce proteinuria more effectively than the standard care, potentially slowing the progression to kidney failure.

From a medical research perspective, the approval could encourage further studies into dual-action drugs and validate the efficacy of this treatment approach. The long-term benefits, such as delaying kidney failure, could translate into reduced healthcare costs associated with kidney disease management. However, it's crucial to monitor post-marketing data to ensure the efficacy and safety profile remains consistent with the study findings.

Market Research Analyst

As sparsentan is poised to become the first of its kind in the European market, it is important to evaluate the market potential and competitive landscape. The Orphan Medicinal Product Designation previously granted by the European Commission underlines the unmet medical need and could provide certain incentives, such as market exclusivity, which may positively influence the stock performance of Travere Therapeutics. The partnership with CSL Vifor could also leverage their established European presence for effective commercialization.

Investors should note that the European Commission's final decision in Q2 2024 will be a critical factor in determining the drug's impact on the market. Moreover, the existing commercialization rights granted to CSL Vifor for other regions like Australia and New Zealand hint at a strategic approach to global market penetration, which could further bolster the company's financial outlook.

Healthcare Economist

The introduction of sparsentan into the European market could have significant economic implications for the healthcare system, particularly in the treatment of rare diseases like IgAN. The cost-effectiveness of sparsentan will be an important consideration, as rare disease treatments often come with high price tags. A reduction in the progression to kidney failure could mean considerable savings in terms of dialysis and transplantation costs. However, it's also necessary to weigh these potential savings against the drug's pricing strategy and reimbursement policies across different European health systems.

Furthermore, the conditional marketing authorization suggests that additional data may be required to confirm the drug's benefit-risk profile, which could entail further investment in clinical research. This conditional status and any subsequent data requirements could influence investor sentiment and the long-term financial sustainability of the drug's market presence.

Committee for Medicinal Products for Human Use (CHMP) recommends approval of the conditional marketing authorization (CMA) for sparsentan for the treatment of IgA nephropathy (IgAN) in Europe

Positive CHMP opinion is based on pivotal phase-III PROTECT study results

European Commission decision is expected in Q2 2024

ST. GALLEN, Switzerland, Feb. 23, 2024 /PRNewswire/ -- CSL Vifor and Travere Therapeutics, Inc., (NASDAQ: TVTX) today announced that the European Medicines Agency's (EMA) CHMP has recommended approval of 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). IgAN is a rare kidney disorder and a leading cause of kidney failure. The CHMP opinion provides the basis for the European Commission's final decision regarding CMA for sparsentan. If approved in Europe, sparsentan will be the first non-immunosuppressive, single-molecule, dual endothelin angiotensin receptor antagonist for the treatment of IgAN.

"The positive CHMP opinion for sparsentan is one step closer to bringing this treatment option to patients in Europe with IgAN, a rare and serious condition that can cause kidney disease," said Emmanuelle Lecomte Brisset, Senior Vice President and Head of Global Regulatory Affairs at CSL. "We look forward to the European Commission decision and to continuing to advance CSL's promise to provide innovative treatments for patients with kidney disease."

"The positive recommendation from the CHMP represents a significant advancement towards the delivery of new treatment options for people living with IgAN, who face the risk of progression to kidney failure and who currently have no approved non-immunosuppressive treatment options. The PROTECT Study is the only head-to-head study in IgAN against a maximally labeled dose of irbesartan, a current standard of care. The study demonstrated treatment with sparsentan resulted in a rapid and sustained reduction in proteinuria and has the potential to preserve kidney function and significantly delay time to kidney failure compared to an active comparator, suggesting long-term benefits in IgAN," said Eric Dube, Ph.D., president and chief executive officer of Travere Therapeutics. "Together with CSL Vifor, we look forward to the European Commission's decision in the second quarter of 2024."

The positive CHMP opinion is based on results from the pivotal phase-III PROTECT study of sparsentan in IgAN, In August 2022, CSL Vifor and Travere Therapeutics announced they had submitted a Marketing Authorization Application (MAA) for CMA to the EMA. The European Commission previously granted Orphan Medicinal Product Designation to sparsentan for the treatment of IgAN.

If approved, sparsentan would receive a CMA in all member states of the European Union, as well as in Iceland, Liechtenstein and Norway. Sparsentan is currently marketed in the U.S. and granted accelerated approval by the U.S. Food and Drug Administration under the brand name FILSPARI^® based on reduction in proteinuria.

In 2021, Travere Therapeutics granted CSL Vifor exclusive commercialization rights for sparsentan in Europe, Australia and New Zealand.

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.

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.

About sparsentan

Sparsentan 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. Sparsentan is currently available in the US and was granted accelerated approval by the US Food and Drug Administration in February 2023 under the brand name FILSPARI^® based on reduction in proteinuria. In September 2023, Travere Therapeutics reported two-year confirmatory results from the Phase 3 PROTECT Study of FILSPARI in IgAN.

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SOURCE Vifor International AG (CSL Vifor)

What is the CHMP's recommendation regarding sparsentan for IgA nephropathy?

The CHMP recommends conditional marketing authorization for sparsentan in Europe for the treatment of IgA nephropathy.

What is the significance of sparsentan if approved in Europe?

If approved, sparsentan will be the first non-immunosuppressive, single-molecule, dual endothelin angiotensin receptor antagonist for the treatment of IgA nephropathy.

What study results led to the positive CHMP opinion for sparsentan?

The positive CHMP opinion is based on results from the pivotal phase-III PROTECT study of sparsentan in IgA nephropathy.

What is the expected timeline for the European Commission's decision on sparsentan?

The European Commission decision on sparsentan is expected in Q2 2024.

What are the potential benefits of sparsentan for IgA nephropathy patients?

Sparsentan has the potential to result in a rapid and sustained reduction in proteinuria, preserve kidney function, and significantly delay time to kidney failure compared to the current standard of care.