Travere Therapeutics is focused on developing innovative therapies for rare kidney diseases, with a particular emphasis on conditions such as focal segmental glomerulosclerosis (FSGS) and IgA nephropathy. One of their key investigational products is Sparsentan, a novel small-molecule candidate currently in Phase 3 development for FSGS. Sparsentan works by selectively targeting the endothelin A receptor and the angiotensin II subtype 1 receptor, which are critical pathways involved in kidney function. Preclinical studies have demonstrated that this dual blockade can reduce proteinuria—a significant marker of kidney disease activity—while also protecting kidney cells and preventing further damage.

In addition to Sparsentan, Travere is also advancing Pegtibatinase (TVT-058) for the treatment of classical homocystinuria, showcasing their commitment to addressing a range of rare metabolic and kidney disorders. The company’s pipeline reflects a robust scientific foundation aimed at setting new standards of care for patients suffering from these challenging conditions. Their ongoing clinical trials, such as the DUPLEX Study for Sparsentan, are designed to evaluate the efficacy and safety of their therapies, with the ultimate goal of improving patient outcomes and quality of life for those affected by rare kidney diseases.

United States

Travere Therapeutics is advancing therapeutic candidates for rare disorders in nephrology, hepatology and metabolism; sponsoring no-cost genetic testing aimed at improving the diagnostic odyssey for children born with cholestasis; partnering with patient advocacy organizations to meet the diverse needs of patients and their families; and ensuring broad access and education for its FDA-approved therapies.

The Company’s pipeline is led by sparsentan, which is being investigated in pivotal Phase 3 clinical trials for rare kidney diseases - focal segmental glomerulosclerosis (FSGS) and IgA nephropathy (IgAN). The DUPLEX Study in FSGS and the PROTECT Study in IgAN are both positioned for topline readouts from the proteinuria endpoints next year. Travere Therapeutics recently expanded its promising pipeline with the addition of OT-58, now called TVT-058, an investigational human enzyme replacement therapy in Phase 1/2 development for the treatment of classical homocystinuria. Travere Therapeutics continues to partner with leaders in patient advocacy and the National Institutes of Health on early research in rare metabolic and hepatic conditions - NGLY1 deficiency and Alagille syndrome.

Genetic Diseases

Kidney Diseases

Rare diseases

Travere Therapeutics

Cystinosis

Kidney disease

The goal of therapy is to reduce urinary cystine concentration below its solubility limit. Tiopronin is an active reducing agent which undergoes thiol-disulfide exchange with cystine to form a mixed disulfide of tiopronin-cysteine. From this reaction, a water-soluble mixed disulfide is formed and the amount of sparingly soluble cystine is reduced.
<a href="https://www.accessdata.fda.gov/drugsatfda_docs/label/2019/211843s000lbl.pdf">Source</a>

THIOLA® EC (tiopronin) 

IgA nephropathy

Sparsentan is an investigational product candidate in Phase 3 clinical development that has a dual mechanism of action combining endothelin receptor type A blockade with angiotensin receptor blockade.&nbsp;Retrophin&nbsp;is developing sparsentan for the treatment of IgAN, as well as for focal segmental glomerulosclerosis (FSGS), rare kidney disorders that often lead to ESRD. In several forms of chronic kidney disease, such as IgAN and FSGS, endothelin receptor blockade has been shown to have an additive beneficial effect on proteinuria in combination with renin-angiotensin blockade via angiotensin receptor blockade or angiotensin converting enzyme inhibitors. Sparsentan has been granted orphan drug designation for the treatment of FSGS by the&nbsp;FDA&nbsp;and&nbsp;European Commission.

FILSPARI (Sparsentan)

Focal segmental glomerulosclerosis

Sparsentan is an investigational product candidate in Phase 3 clinical development that has a dual mechanism of action combining endothelin receptor type A blockade with angiotensin receptor blockade.&nbsp;Retrophin&nbsp;is developing sparsentan for the treatment of FSGS, as well as for IgA nephropathy (IgAN), rare kidney disorders that often lead to ESRD. In several forms of chronic kidney disease, such as FSGS and IgAN, endothelin receptor blockade has been shown to have an additive beneficial effect on proteinuria in combination with renin-angiotensin blockade via angiotensin receptor blockade or angiotensin converting enzyme inhibitors. Sparsentan has been granted orphan drug designation for the treatment of FSGS by the&nbsp;FDA&nbsp;and&nbsp;European Commission.

Homocystinuria

TVT-058 &nbsp;is a PEGylated, recombinant enzyme replacement candidate designed to &nbsp;address the underlying cause of classical HCU — a deficiency in the &nbsp;naturally occurring enzyme cystathionine beta synthase (CBS). The goal &nbsp;of the TVT-058 program is to develop a well-tolerated, safe and &nbsp;effective treatment that sustainably replaces CBS to reduce levels of &nbsp;homocysteine.
<a href="https://travere.com/our-pipeline/tvt-058/">Source</a>

Pegtibatinase (TVT-058)

Cerebrotendinous xanthomatosis

CTX &nbsp;is a bile acid synthesis disorder resulting in the deficiency of an &nbsp;enzyme necessary to convert cholesterol into the bile acid called &nbsp;chenodeoxycholic acid (CDCA). As a result the body is unable to break &nbsp;down cholesterol properly, causing toxins (e.g., cholestanol and bile &nbsp;alcohols) to build up over time. Affected individuals can experience &nbsp;neonatal cholestasis (jaundice or bile flow interruption), chronic &nbsp;diarrhea, the development of cataracts in both eyes before the age of &nbsp;18, development of tendon xanthomas (fatty deposits in the tendons) &nbsp;during teenage years or later, and neurologic deterioration. There are &nbsp;no medications approved for the treatment of the disease.
Our &nbsp;investigational approach is to evaluate the use of Chenodal as a direct &nbsp;replacement therapy option for the restoration of physiologic bile &nbsp;acids and the regulation of cholestanol and bile alcohols compared to &nbsp;placebo.
<a href="https://travere.com/our-pipeline/chenodeoxycholic-acid/">Source</a>

Travere Therapeutics

Company Details