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Travere Therapeutics

Latest, April 13, 2026Filspari (sparsentan) became the first FDA-approved treatment for FSGS on April 13, 2026, for adults and children 8 and older without nephrotic syndrome, adding to its full approval for IgA nephropathy. Filspari drug page with FDA sources

Travere Therapeutics works on 6 rare diseases tracked on Trial Friend, including Alport Syndrome, Cerebrotendinous Xanthomatosis, Focal Segmental Glomerulosclerosis and 3 more, with 6 active clinical trials (4 recruiting) and 2 FDA-approved rare disease drugs.

Travere Therapeutics is a San Diego biotech focused on rare kidney and metabolic diseases. The company was founded in 2011 as Retrophin and rebranded as Travere in November 2020. Travere trades on the NASDAQ under the ticker TVTX and built its current pipeline around a concrete question. What happens to patients with rare kidney diseases when their condition has no approved treatment?

That question came to a head with focal segmental glomerulosclerosis (FSGS), a rare kidney disease that scars the filtering units of the kidneys and leads many patients toward dialysis or transplant. Despite affecting tens of thousands of Americans, FSGS had never had an FDA-approved drug, and that changed on April 13, 2026, when the FDA granted Travere's drug Filspari (sparsentan) full approval for FSGS in adults and pediatric patients aged 8 and older who do not have nephrotic syndrome, making it the first and only approved medicine for the disease. The approval was based on the Phase 3 DUPLEX study, where 371 patients were randomized between sparsentan and irbesartan, a standard blood pressure drug used off-label in FSGS. Patients on sparsentan saw a 46% reduction in protein leakage from the kidneys at week 108, compared to 30% on irbesartan. The trial found no significant difference between the 2 drugs in how fast kidney function declined, and the FDA approved Filspari for FSGS to reduce protein leakage in people 8 and older who do not have nephrotic syndrome.

Filspari was already approved in IgA nephropathy, a different rare kidney disease where the immune system inappropriately attacks the kidneys. Filspari received full FDA approval for IgA nephropathy in September 2024 and is one of several new drugs that have recently changed the IgAN treatment landscape. Travere also markets THIOLA EC (tiopronin) for cystinuria, a rare disease that causes recurrent kidney stones starting in childhood.

The lead pipeline asset is pegtibatinase for classical homocystinuria, a rare metabolic disorder where the body cannot properly process the amino acid homocysteine. Pegtibatinase is in a pivotal Phase 3 trial called HARMONY and has FDA Breakthrough Therapy designation. Enrollment restarted in early 2026 after a pause to scale up manufacturing, and Travere expects topline data in the second half of 2027. Across the portfolio in 2025, Travere reported $410 million in net product sales, with Filspari generating $322 million of that in U.S. revenue.

Type
Rare Disease Specialist
Ticker
TVTX
Headquarters
San Diego, United States
Founded
2011
6
Active Rare Disease Trials
2
Approved Rare Disease Drugs
6
Rare Diseases in Portfolio
15
Years Active

Travere Therapeutics Drug Pipeline

Travere Therapeutics has 6 active clinical trials across 3 development stages, with 4 currently recruiting participants. Clinical trials advance through phases: Phase 1 tests safety in a small group, Phase 2 evaluates effectiveness and side effects, Phase 3 confirms benefit in a larger population, and Phase 4 monitors long-term safety after FDA approval.

Note: This pipeline counts the active and recruiting trials listed on this page, from ClinicalTrials.gov. For companies that work outside rare disease, we list only trials for rare diseases we track, so the company's full pipeline may be larger.

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3
Phase 23 trials
2
Phase 32 trials
Recruiting
Active Not Recruiting
1
Phase 4 / Post-Market1 trial
Proteinuria+3 more
Active Not Recruiting

Travere Therapeutics Clinical Trials (6)

Active and recruiting clinical trials sponsored by Travere Therapeutics, sourced live from ClinicalTrials.gov. Each trial card shows the study phase, current recruitment status, conditions under investigation, study locations, eligibility criteria, and a direct link to the full ClinicalTrials.gov record. You can also download a one-page PDF summary to share with your doctor.

Note: Recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator.

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RECRUITINGPHASE3Recently updatedNCT06247085

Pegtibatinase as a Treatment for Participants With Classical Homocystinuria (HCU) Receiving Standard of Care Treatment

Intervention: Pegtibatinase, Placebo

Researchers are looking for a better way to treat people living with classical homocystinuria (HCU), a rare inherited condition. It is caused by changes in the cystathionine beta-synthase (or "CBS") gene and prevents an enzyme from working correctly in the body. This enzyme helps break down homocysteine, which is produced when the body processes methionine from protein-containing foods. This helps keep homocysteine and methionine at healthy levels. When this enzyme is not working, homocysteine and methionine build up in the blood and then spread into different tissues of the body and stop these body tissues from working normally. People living with HCU can experience problems with their vision, bones, blood vessels, thinking, learning, and memory. Available treatments for HCU include a low-methionine diet (typically achieved by limiting protein-containing foods), a methionine-free protein supplement to help meet daily protein needs, and betaine (Cystadane®). These treatments are either not sufficient or are hard to take for many patients. Pegtibatinase was designed by researchers to act like the CBS enzyme, which is missing or not working properly in people living with HCU. Researchers are studying whether adding pegtibatinase to standard treatment can lower homocysteine levels. Researchers want to learn how safe and effective pegtibatinase is, how it moves through and affects the body, and how it may affect the daily lives of people with HCU. The study plans to enroll participants from the US, Australia, Belgium, France, Germany, Ireland, Italy, Poland, Portugal, Qatar, Saudi Arabia, Spain, Turkey, and the United Kingdom. Many study visits may happen at home, depending on where you live and local regulations. Some visits may include a home nurse and telemedicine appointments with the study doctor.

Ages 12 Years - 65 Years52 locations
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RECRUITINGPHASE1, PHASE2Recently updatedNCT03406611

Pegtibatinase as a Treatment for Patients With Classical Homocystinuria (HCU) (Also Known as the COMPOSE Study)

Intervention: Pegtibatinase, Placebo

Researchers are looking for a better way to treat people who have classical homocystinuria (HCU), a rare condition that is passed down by parents (or "genetic condition"). It is caused by changes in the cystathionine beta-synthase (or "CBS") gene and prevents an enzyme from working correctly in the body. This enzyme breaks down a substance called homocysteine (from dietary methionine found in protein) and keeps both homocysteine and methionine at normal levels. When this enzyme is not working, homocysteine and methionine build up in the blood, which spreads into different tissues of the body and stops these body tissues from working normally. People with HCU can experience problems with vision, bones, blood vessels, and cognitive function (the ability to think, learn, and remember). Treatments available for HCU, such as a low protein diet and betaine (Cystadane®), help reduce homocysteine levels. The diet is a low methionine diet and a methionine-free protein supplement (a product that provides extra protein to help meet daily protein needs). These treatments are either not sufficient or are hard to take for many patients. Pegtibatinase was developed by scientists to be a version of the CBS enzyme that can be given to people with HCU. Researchers believe that giving pegtibatinase to people with HCU already getting medical treatment (or "standard of care") may reduce their homocysteine levels. This study is split into 7 different groups getting different amounts of drug. The first 6 groups have already finished the study. Group 7 plans to enroll participants from the US (virtual and in-person), France, and Qatar.

Ages 5 Years - 65 Years12 locations
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RECRUITINGPHASE2Updated a few months agoNCT04663204

A Study of the Safety and Activity of Sparsentan for the Treatment of Patients With Immunoglobulin A Nephropathy

Intervention: Sparsentan

Immunoglobulin A NephropathyKidney DiseasesGlomerulonephritis, IGAGlomerulonephritisAutoimmune Diseases

To determine the nephroprotective potential of treatment with sparsentan in (1: Cohort A) patients newly-diagnosed with immunoglobulin A nephropathy (IgAN) (ie, incident patients) who have not received prior angiotensin-converting enzyme inhibitor (ACEI) or angiotensin receptor blocker (ARB) therapy, and in (2: Cohort B) patients with recurrent IgAN following kidney transplantation.

Ages 18 Years+6 locations
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RECRUITINGPHASE2Updated a few months agoNCT05003986

Study of Sparsentan Treatment in Pediatrics With Proteinuric Glomerular Diseases

Intervention: Sparsentan, Sparsentan, Sparsentan

To evaluate the safety, efficacy and tolerability of sparsentan oral suspension and tablets, and assess changes in proteinuria after once-daily dosing over 108 weeks.

Ages 1 Year - 17 Years47 locations
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ACTIVE NOT RECRUITINGPHASE4Updated a few months agoNCT07219121

Sparsentan in Posttransplant Immunoglobulin A Nephropathy or Focal Segmental Glomerulosclerosis

Intervention: Sparsentan

ProteinuriaImmunoglobulin A (IgA) NephropathyFocal Segmental GlomerulosclerosisKidney Transplant

To evaluate the safety and efficacy of sparsentan tablets for the treatment of patients with proteinuria after kidney transplantation with once-daily dosing for 36 weeks.

Ages 18 Years+8 locations
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ACTIVE NOT RECRUITINGPHASE3Updated a few months agoNCT03762850

A Study of the Effect and Safety of Sparsentan in the Treatment of Patients With IgA Nephropathy

Intervention: sparsentan, irbesartan, Dapagliflozin

To determine the long-term (approximately 2 years) nephroprotective potential of treatment with sparsentan as compared to an angiotensin receptor blocker in patients with immunoglobulin A nephropathy (IgAN).

Ages 18 Years+162 locations
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Travere Therapeutics FDA-Approved Drugs (2)

Medications developed or marketed by Travere Therapeutics that have received U.S. Food and Drug Administration approval. Drug data is sourced from the openFDA database and includes brand names, generic names, approval dates, and matched rare disease indications. Where a drug treats a condition covered by Trial Friend, the disease name links directly to that disease page.

Drug NameBrand NameRare DiseasesApproval Date
CHENODIOL
Bile Acid [EPC]
Chenodal
oral
—Oct 22, 2009
SPARSENTAN
Endothelin Receptor Antagonist [EPC]
FILSPARI
oral
Feb 17, 2023

Travere Therapeutics Trial Locations

Travere Therapeutics clinical trials are running at 287 sites in 24 countries. Click any country to drill down by state, city, and individual research facility. Proximity to a trial site is one of the most important factors in deciding whether to participate.

United States
94▼
United Kingdom
35▼
Spain
22▼
Italy
20▼
Germany
16▼
Australia
13▼
France
12▼
Poland
12▼
South Korea
10▼
Portugal
9▼
Belgium
8▼
Hong Kong
5▼

Rare Disease Focus Areas (6)

Diseases targeted by Travere Therapeutics's clinical trial and drug development programs

Alport SyndromeKidney & Renal

Alport Syndrome is a genetic disorder that causes progressive damage to the kidneys, ears, and eyes due to defects in a type of collagen that provides structure and flexibility to tissues. The conditi...

Prevalence: Approximately 1 in 5,000 to 10,000 people worldwide
Cerebrotendinous XanthomatosisMetabolic & Lysosomal

Cerebrotendinous xanthomatosis (CTX) is a rare inherited metabolic disorder caused by mutations in the CYP27A1 gene, which produces an enzyme essential for converting cholesterol into bile acids. The ...

Prevalence: Prevalence estimates vary widely by population. Genetic-frequency analyses suggest CTX may affect roughly 1 in 70,000 Americans, with somewhat higher rates in South and East Asian populations, but only around 300 individuals have been clinically identified worldwide, indicating substantial under-diagnosis. The average age at clinical diagnosis is 35 years, and the typical diagnostic delay from symptom onset is approximately 16 years.
Focal Segmental GlomerulosclerosisKidney & Renal

Focal Segmental Glomerulosclerosis (FSGS) is a kidney disease characterized by scarring of some glomeruli, the tiny filtering units that remove waste from the blood. The disease can develop on its own...

Prevalence: Approximately 7% of all kidney biopsies in the United States show FSGS; incidence is 0.2-1.7 cases per 100,000 person-years
HomocystinuriaMetabolic & Lysosomal

Homocystinuria is a rare inherited metabolic disorder characterized by elevated homocysteine levels in blood and urine. 3 biochemically distinct types exist; the most common involves cystathionine bet...

Prevalence: 1 in 340,000 births (cystathionine beta-synthase deficiency)
IgA NephropathyKidney & Renal

IgA nephropathy is the most common primary glomerulonephritis worldwide, caused by abnormal IgA1 antibodies depositing in the kidney's mesangium, activating complement pathways and triggering inflamma...

Prevalence: About 2 new cases per 100,000 people each year in the U.S., and an estimated 199,000 to 208,000 Americans were living with IgA nephropathy in 2021 (Otsuka-funded study, Clinical Nephrology, 2025). About 1 in 10 kidney biopsies in the U.S. show IgA nephropathy (NIDDK). Significantly more common in East Asian and Pacific Islander populations, with reported incidence up to 4 times higher.
IgA Vasculitis with NephritisKidney & Renal

IgA vasculitis is a small-vessel vasculitis characterized by IgA immune complex deposition in kidneys and other organs. The condition manifests with purpura, arthritis, and glomerulonephritis. Most pa...

Prevalence: About 3 to 27 new cases per 100,000 children each year; the most common vasculitis of childhood

Patient Resources

Organizations and resources related to Travere Therapeutics's rare disease focus areas

Frequently Asked Questions About Travere Therapeutics

Common questions about Travere Therapeutics's rare disease programs, clinical trials, and treatments.