Travere Therapeutics
Travere Therapeutics works on 6 rare diseases tracked on Trial Friend, including Alport Syndrome, Cerebrotendinous Xanthomatosis, Focal Segmental Glomerulosclerosis and 3 more, with 6 recruiting clinical trials 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, with slower kidney function decline as well.
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 pegtibatase for classical homocystinuria, a rare metabolic disorder where the body cannot properly process the amino acid homocysteine. Pegtibatase is in a pivotal Phase 3 trial called HARMONY and has FDA Breakthrough Therapy designation. Enrollment resumed in February 2026 after a brief manufacturing scale-up pause, with topline data expected in 2026. Across the portfolio in 2025, Travere reported $410 million in net product sales, with Filspari generating $322 million of that in U.S. revenue.
Travere Therapeutics Drug Pipeline
Travere Therapeutics has 6 active clinical trials across 3 development stages, with 6 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 includes all of Travere Therapeutics's active interventional trials, not only those targeting rare diseases. We show the full pipeline because a company's broader research activity, therapeutic expertise, and development infrastructure directly shape its ability to advance rare disease programs. A strong overall pipeline often signals deeper clinical operations, faster enrollment capabilities, and greater commitment to bringing new treatments to patients.
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.
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 Name | Brand Name | Rare Diseases | Approval 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.
Rare Disease Focus Areas (6)
Diseases targeted by Travere Therapeutics's clinical trial and drug development programs
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...
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 ...
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...
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...
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...
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...
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.