This section highlights FDA-related milestones and regulatory updates for drugs developed by Travere Therapeutics (TVTX).
Over the past two years, Travere Therapeutics has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
FILSPARI, Pegtibatinase, and Sparsentan. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Select a button below to view the list of FDA events for that drug.
FILSPARI FDA Regulatory Timeline and Events
FILSPARI is a drug developed by Travere Therapeutics for the following indication: Endothelin and angiotensin II receptor antagonist indicated to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, generally a UPCR ≥1.5 g/g.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- FILSPARI
- Announced Date:
- April 14, 2026
- Indication:
- Endothelin and angiotensin II receptor antagonist indicated to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, generally a UPCR ≥1.5 g/g.
Announcement
Ligand Pharmaceuticals announced that its partner Travere Therapeutics, Inc has received approval from the U.S. Food and Drug Administration (FDA) for FILSPARI® (sparsentan) to reduce proteinuria in adult and pediatric patients aged 8 years and older with focal segmental glomerulosclerosis (FSGS) without nephrotic syndrome..
AI Summary
Ligand Pharmaceuticals announced that its partner Travere Therapeutics has received FDA approval for FILSPARI® (sparsentan) to reduce proteinuria in adult and pediatric patients aged 8 years and older with focal segmental glomerulosclerosis (FSGS) without nephrotic syndrome. The approval covers patients whose kidney disease is driven largely by stress on the glomeruli, which can trigger inflammation and scarring and lead to worsening kidney function.
FILSPARI works with a dual mechanism of action, targeting endothelin A and angiotensin II receptors to help protect the kidney and reduce damage. Ligand said the approval provides a new treatment option for people with this rare and serious kidney disorder.
Under its agreement with Travere, Ligand is entitled to a 9% royalty on worldwide net sales of FILSPARI. Ligand’s CEO congratulated Travere and said the approval offers new hope for patients and potential long-term royalty growth for Ligand.
Read Announcement- Drug:
- FILSPARI
- Announced Date:
- April 13, 2026
- Indication:
- Endothelin and angiotensin II receptor antagonist indicated to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, generally a UPCR ≥1.5 g/g.
Announcement
Travere Therapeutics, Inc announced that the U.S. Food and Drug Administration (FDA) has approved FILSPARI® (sparsentan) to reduce proteinuria in adult and pediatric patients aged 8 years and older with focal segmental glomerulosclerosis (FSGS) without nephrotic syndrome..
AI Summary
Travere Therapeutics announced FDA approval of FILSPARI (sparsentan) to reduce proteinuria in adults and pediatric patients aged 8 years and older with focal segmental glomerulosclerosis (FSGS) who do not have nephrotic syndrome. The new indication expands the U.S. addressable population to more than 100,000 people, including over 30,000 patients with FSGS, and the label aligns with KDIGO clinical practice guidelines.
In FSGS without nephrotic syndrome, kidney injury is driven by stress on the glomeruli and activation of inflammatory and scarring pathways. FILSPARI’s dual mechanism targets endothelin A and angiotensin II receptors, which is believed to help protect the kidney and reduce proteinuria.
FILSPARI has boxed warnings for hepatotoxicity and embryo‑fetal toxicity and is available only through a REMS program. Measure transaminases and bilirubin before treatment and every three months; avoid use if baseline aminotransferases are >3× ULN. It is contraindicated in pregnancy and with ARBs, ERAs, or aliskiren. Common side effects in FSGS include peripheral edema, hypotension, hyperkalemia, dizziness, and anemia. Avoid strong CYP3A inducers and monitor P‑gp substrates.
Travere will host a conference call on April 13, 2026, at 6:45 p.m. ET.
Read Announcement- Drug:
- FILSPARI
- Announced Date:
- January 13, 2026
- Target Action Date:
- April 13, 2026
- Indication:
- Endothelin and angiotensin II receptor antagonist indicated to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, generally a UPCR ≥1.5 g/g.
Announcement
Travere Therapeutics, Inc announced that The new Prescription Drug User Fee Act (PDUFA) target action date is April 13, 2026.
AI Summary
Travere Therapeutics announced the U.S. Food and Drug Administration has extended its review of the supplemental New Drug Application (sNDA) for FILSPARI in focal segmental glomerulosclerosis (FSGS). The new PDUFA target action date is April 13, 2026. The extension followed Travere’s submission of responses that the FDA classified as a Major Amendment, which moved the review timeline. The agency did not request additional safety or manufacturing information.
Travere said it remains engaged with the FDA and will use the extension to continue dialogue and supply any needed information. The company is also preparing for a potential commercial launch so it can act quickly if approved.
If approved, FILSPARI would be the first medicine for FSGS, a rare kidney disease marked by protein in the urine and progressive kidney failure. It is an oral, non‑immunosuppressive therapy that targets podocyte injury.
Read Announcement- Drug:
- FILSPARI
- Announced Date:
- January 13, 2026
- Indication:
- Endothelin and angiotensin II receptor antagonist indicated to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, generally a UPCR ≥1.5 g/g.
Announcement
Travere Therapeutics, Inc., announced that today the U.S. Food and Drug Administration (FDA) has extended the review timeline of its supplemental New Drug Application (sNDA) for FILSPARI® (sparsentan) in focal segmental glomerulosclerosis (FSGS).
AI Summary
Travere Therapeutics announced that the U.S. Food and Drug Administration has extended the review timeline for its supplemental New Drug Application (sNDA) for FILSPARI® (sparsentan) in focal segmental glomerulosclerosis (FSGS). The new Prescription Drug User Fee Act (PDUFA) target action date is April 13, 2026.
The extension follows Travere’s submission of FDA-requested responses to further characterize FILSPARI’s clinical benefit. The FDA determined those responses constituted a Major Amendment, which triggered the new action date. The agency did not request any additional information about FILSPARI’s safety or manufacturing.
Travere said it will continue commercial launch preparations while working with the FDA during the extension period. If approved, FILSPARI could become the first medication indicated specifically for FSGS, a rare kidney disorder driven by proteinuria that can lead to progressive kidney damage and failure. The company said it remains focused on bringing a new treatment option to patients with urgent need.
Read Announcement- Drug:
- FILSPARI
- Announced Date:
- November 6, 2025
- Indication:
- Endothelin and angiotensin II receptor antagonist indicated to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, generally a UPCR ≥1.5 g/g.
Announcement
Travere Therapeutics, Inc announced new data from the Phase 3 DUPLEX Study demonstrating that patients with focal segmental glomerulosclerosis (FSGS) treated with FILSPARI® (sparsentan) were significantly more likely to reach proteinuria levels below 0.7 g/g compared to those receiving the maximum labeled dose of irbesartan, and achievement of this threshold correlated with reduced risk of kidney failure.
AI Summary
Travere Therapeutics reported new Phase 3 DUPLEX Study results showing that patients with focal segmental glomerulosclerosis (FSGS) treated with FILSPARI® (sparsentan) were significantly more likely to lower their urine protein-to-creatinine ratio (UPCR) below 0.7 g/g compared with those on the highest dose of irbesartan. At any time during the study, 37.5% of FILSPARI patients reached this target versus 21.4% with irbesartan, and at week 108, rates were 19.0% versus 11.2%, respectively.
Reaching UPCR below 0.7 g/g was linked to fewer kidney failure events. In DUPLEX, patients who hit the threshold had a 3.6% rate of kidney failure versus 11.2% for those who did not. A RaDaR registry analysis showed a 60-month follow-up hazard ratio of 0.14 for patients who reached the threshold at 24 months, and 0.27 for those who reached it at any time over two years.
These findings reinforce the PARASOL Project conclusion that reducing proteinuria below 0.7 g/g is key to long-term kidney preservation in FSGS.
Read Announcement- Drug:
- FILSPARI
- Announced Date:
- September 10, 2025
- Indication:
- Endothelin and angiotensin II receptor antagonist indicated to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, generally a UPCR ≥1.5 g/g.
Announcement
Travere Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has informed the Company that following further review of the supplemental New Drug Application (sNDA) for FILSPARI® (sparsentan) in focal segmental glomerulosclerosis (FSGS), an advisory committee is no longer needed.
AI Summary
Travere Therapeutics, Inc. announced that the U.S. Food and Drug Administration recently informed the company that after further review of its supplemental New Drug Application (sNDA) for FILSPARI® (sparsentan) in focal segmental glomerulosclerosis (FSGS), an advisory committee is no longer needed. The FDA continues its review, with a Prescription Drug User Fee Act (PDUFA) target action date of January 13, 2026.
FSGS is a rare and serious kidney disorder that causes protein to leak into the urine, leading to scarring and progressive kidney function loss. FILSPARI is an oral, non-immunosuppressive therapy designed to protect podocytes—the cells that filter blood in the kidney—and reduce proteinuria, the main driver of FSGS progression. If approved, FILSPARI would become the first medication specifically indicated for FSGS.
Eric Dube, Ph.D., president and CEO of Travere, said the company is pleased with the progress of the sNDA and is preparing for a potential commercial launch in January 2026. Travere will continue to support the FDA’s review in hopes of delivering a new treatment option for patients with this challenging disease.
Read Announcement- Drug:
- FILSPARI
- Announced Date:
- June 11, 2025
- Indication:
- Endothelin and angiotensin II receptor antagonist indicated to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, generally a UPCR ≥1.5 g/g.
Announcement
Travere Therapeutics, Inc. announced that the Company will present three abstracts on the effect of FILSPARI (sparsentan) in rare kidney disease at the upcoming International Podocyte Conference in Hamburg, Germany, June 10-13, 2025.
AI Summary
Travere Therapeutics, Inc. announced that it will present three abstracts at the International Podocyte Conference in Hamburg, Germany, from June 10–13, 2025. These presentations will highlight the effect of FILSPARI (sparsentan) in rare kidney diseases, especially IgA nephropathy (IgAN). New data from the Phase 2 SPARTAN Study shows that FILSPARI can lead to rapid and lasting reductions in certain biomarkers, suggesting both anti-inflammatory and antifibrotic benefits. This information indicates that FILSPARI may have disease-modifying effects by slowing the progression of kidney damage in patients with IgAN. Travere’s efforts to share these findings at the conference underline the company’s commitment to advancing treatment options for rare kidney diseases and informing the medical community about potential new therapies.
Read Announcement- Drug:
- FILSPARI
- Announced Date:
- June 3, 2025
- Indication:
- Endothelin and angiotensin II receptor antagonist indicated to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, generally a UPCR ≥1.5 g/g.
Announcement
Travere Therapeutics, Inc. announced that the Company will present seven abstracts at the upcoming European Renal Association (ERA) Congress in Vienna, Austria, June 4-7.
AI Summary
Travere Therapeutics has announced that it will present seven abstracts at the upcoming European Renal Association Congress in Vienna, Austria, from June 4-7. The abstracts will focus on new data involving FILSPARI in treating IgA nephropathy. One key study, the Phase 2 SPARTACUS trial, showed significant improvements when patients with IgA nephropathy switched from renin-angiotensin system inhibitors to FILSPARI while continuing stable SGLT2 inhibitor therapy. Additionally, data from the open-label extension of the Phase 3 PROTECT study will demonstrate notable reductions in proteinuria for those who transitioned from maximum label irbesartan treatment to FILSPARI. Travere’s presentations also include mechanistic insights suggesting that FILSPARI may protect against IgA deposition in the kidney, further supporting its potential as a foundational therapy for rare kidney diseases.
Read Announcement- Drug:
- FILSPARI
- Announced Date:
- May 15, 2025
- Indication:
- Endothelin and angiotensin II receptor antagonist indicated to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, generally a UPCR ≥1.5 g/g.
Announcement
Travere Therapeutics, Inc announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) for traditional approval of FILSPARI (sparsentan) for the treatment of focal segmental glomerulosclerosis (FSGS).
AI Summary
Travere Therapeutics announced that the FDA has accepted its supplemental New Drug Application (sNDA) for traditional approval of FILSPARI (sparsentan) for treating focal segmental glomerulosclerosis (FSGS), a rare kidney disease that can lead to kidney failure. The FDA has set a PDUFA target action date of January 13, 2026, and is expected to hold an advisory committee meeting to discuss the application. FILSPARI is an oral, non-immunosuppressive medication that works by blocking specific receptors involved in kidney damage. If approved, it would be the first FDA-approved treatment for FSGS, offering a new therapeutic option for patients who have long needed effective treatment. This step reflects Travere’s commitment to transforming care for patients with rare kidney diseases and highlights their focus on addressing significant unmet medical needs in the FSGS community.
Read Announcement- Drug:
- FILSPARI
- Announced Date:
- April 29, 2025
- Indication:
- Endothelin and angiotensin II receptor antagonist indicated to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, generally a UPCR ≥1.5 g/g.
Announcement
CSL Vifor and Travere Therapeutics, Inc. announce that the European Commission has approved the conversion of the conditional marketing approval (CMA) into a standard marketing authorization (MA) for FILSPARI for the treatment of adults with primary IgA nephropathy with a urine protein excretion ≥1.0 g/day (or urine protein-to-creatinine ratio ≥0.75 g/g).
AI Summary
CSL Vifor and Travere Therapeutics announced that the European Commission has converted the conditional marketing approval of FILSPARI into a standard marketing authorization. This decision allows the drug to be available across the European Union as well as in Iceland, Liechtenstein, and Norway. FILSPARI is approved for treating adults with primary IgA nephropathy who exhibit significant proteinuria (urine protein excretion ≥1.0 g/day or a urine protein-to-creatinine ratio ≥0.75 g/g).
The approval follows a positive recommendation from the Committee for Medicinal Products for Human Use and is based on the complete data set from the pivotal phase-III PROTECT study. The study showed that FILSPARI significantly reduced proteinuria and slowed the decline in kidney function compared to a standard treatment. Both companies emphasized their commitment to improving patient outcomes and expanding access to this innovative therapy for IgA nephropathy.
Read Announcement- Drug:
- FILSPARI
- Announced Date:
- March 31, 2025
- Indication:
- Endothelin and angiotensin II receptor antagonist indicated to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, generally a UPCR ≥1.5 g/g.
Announcement
Travere Therapeutics, Inc., announced that the Company will present three abstracts, including one late-breaking oral presentation, at the upcoming National Kidney Foundation (NKF) Spring Clinical Meetings 2025, taking place April 10-13 in Boston, MA.
AI Summary
Travere Therapeutics, Inc. announced that it will present three abstracts at the National Kidney Foundation (NKF) Spring Clinical Meetings 2025 in Boston, MA, from April 10-13. One presentation, a late-breaking oral session, will focus on new analyses from the Phase 3 DUPLEX Study of FILSPARI® (sparsentan) in patients with focal segmental glomerulosclerosis (FSGS). The data show that patients treated with FILSPARI achieved partial or complete proteinuria remission earlier and more frequently than those treated with irbesartan, with lower rates of kidney failure among responders.
In addition, Travere will share findings on FILSPARI's role as a foundational therapy in IgA nephropathy, including interim results from the Phase 2 SPARTAN Study and real-world case studies across diverse patient backgrounds. These presentations highlight the potential of FILSPARI in improving kidney outcomes in rare kidney diseases.
Read Announcement- Drug:
- FILSPARI
- Announced Date:
- February 11, 2025
- Target Action Date:
- Q1 2025
- Estimated Target Date Range:
- January 1, 2025 - March 31, 2025
- Indication:
- Endothelin and angiotensin II receptor antagonist indicated to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, generally a UPCR ≥1.5 g/g.
Announcement
Travere Therapeutics, Inc. announced that The sNDA will be based on existing data from the Phase 3 DUPLEX and Phase 2 DUET studies of FILSPARI and is expected to be submitted around the end of the first quarter of 2025.
AI Summary
Travere Therapeutics, Inc. announced that it plans to submit a supplemental New Drug Application (sNDA) for FILSPARI around the end of the first quarter of 2025. This submission will be based solely on existing data from two key studies: the Phase 3 DUPLEX study and the Phase 2 DUET study, both of which evaluated FILSPARI in patients with focal segmental glomerulosclerosis (FSGS). If approved, FILSPARI could become the first and only medicine specifically indicated for FSGS, a rare kidney disorder that currently lacks approved treatment options. By using robust data from these two of the largest interventional studies in FSGS, Travere aims to provide a new therapeutic option for a patient population in urgent need. This move underscores the company's commitment to addressing rare kidney diseases through innovative clinical research.
Read Announcement- Drug:
- FILSPARI
- Announced Date:
- February 11, 2025
- Indication:
- Endothelin and angiotensin II receptor antagonist indicated to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, generally a UPCR ≥1.5 g/g.
Announcement
Travere Therapeutics, Inc. announced the Company has completed its Type C meeting with the U.S. Food and Drug Administration (FDA) and plans to submit a supplemental New Drug Application (sNDA) seeking traditional approval of FILSPARI for focal segmental glomerulosclerosis (FSGS).
AI Summary
Travere Therapeutics, Inc. announced that it has completed a Type C meeting with the U.S. Food and Drug Administration (FDA) and is moving forward with a supplemental New Drug Application (sNDA) for FILSPARI. The submission, expected to be filed around the end of the first quarter of 2025, aims to gain traditional approval for FILSPARI as a treatment for focal segmental glomerulosclerosis (FSGS), a rare kidney disorder. The sNDA will be supported by results from the Phase 3 DUPLEX and Phase 2 DUET studies, which have provided valuable data in the FSGS patient population. FILSPARI could become the first approved therapy for FSGS if the sNDA is successful, addressing an urgent need for treatment options for more than 40,000 patients in the U.S. who suffer from this progressive kidney condition.
Read Announcement- Drug:
- FILSPARI
- Announced Date:
- September 6, 2024
- Indication:
- Endothelin and angiotensin II receptor antagonist indicated to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, generally a UPCR ≥1.5 g/g.
Announcement
Ligand Pharmaceuticals Incorporated announced that its partner Travere Therapeutics, Inc has received full approval from the U.S. Food and Drug Administration (FDA) for FILSPARI® (sparsentan) to slow kidney function decline in adults with primary IgAN who are at risk of disease progression. Ligand is entitled to milestone payments and a 9% royalty on worldwide net sales of FILSPARI.
AI Summary
Ligand Pharmaceuticals announced that its partner, Travere Therapeutics, has received full FDA approval for FILSPARI® (sparsentan) to slow the decline of kidney function in adults with primary IgA nephropathy (IgAN) who are at risk of disease progression. This marks a significant advancement for patients suffering from this rare kidney disease.
The approval is supported by positive long-term data from the PROTECT Study, which showed that FILSPARI significantly slows kidney function decline compared to an active comparator. FILSPARI is unique as an oral, once-daily, non-immunosuppressive treatment that targets key pathways involved in IgAN progression. Additionally, Ligand is set to benefit from the partnership, receiving milestone payments and a 9% royalty on worldwide net sales of FILSPARI.
Read Announcement- Drug:
- FILSPARI
- Announced Date:
- September 5, 2024
- Indication:
- Endothelin and angiotensin II receptor antagonist indicated to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, generally a UPCR ≥1.5 g/g.
Announcement
Travere Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted full approval to FILSPARI® (sparsentan) to slow kidney function decline in adults with primary IgAN who are at risk of disease progression.,
AI Summary
Travere Therapeutics announced that the FDA has granted full approval to FILSPARI® (sparsentan) to slow kidney function decline in adults with primary IgA nephropathy (IgAN) who are at risk for disease progression. This milestone follows the successful PROTECT Study, which showed that FILSPARI significantly reduced the rate of kidney function decline over a two-year period compared to irbesartan. The study demonstrated a statistically significant treatment benefit by preserving kidney function and providing a durable reduction in proteinuria. As a once-daily, oral, non-immunosuppressive medication, FILSPARI offers a promising treatment option that targets key pathways in IgAN, potentially setting a new standard of care for these patients. The updated FDA label reflects these long-term benefits, giving physicians greater confidence in prescribing FILSPARI for patients needing effective therapy to protect kidney health.
Read Announcement- Drug:
- FILSPARI
- Announced Date:
- May 6, 2024
- Target Action Date:
- September 5, 2024
- Indication:
- Endothelin and angiotensin II receptor antagonist indicated to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, generally a UPCR ≥1.5 g/g.
Announcement
Travere Therapeutics, Inc. announced that PDUFA target action date of September 5, 2024
AI Summary
Travere Therapeutics recently announced that the FDA has granted Priority Review for its supplemental New Drug Application (sNDA) to convert FILSPARI® (sparsentan) from accelerated approval to full approval for the treatment of IgA nephropathy (IgAN) in the U.S. The FDA has set a PDUFA target action date of September 5, 2024, which indicates that a decision on the application is expected by that time.
This milestone is a significant step forward for the company, as full approval could expand access to FILSPARI for patients with IgAN. The review process reflects the FDA’s commitment to evaluating the potential benefits of converting FILSPARI’s regulatory status. Travere’s efforts to achieve full approval are part of its broader strategy to enhance its treatment offerings for rare diseases and address the unmet needs of patients dealing with IgAN.
Read Announcement- Drug:
- FILSPARI
- Announced Date:
- May 6, 2024
- Indication:
- Endothelin and angiotensin II receptor antagonist indicated to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, generally a UPCR ≥1.5 g/g.
Announcement
Travere Therapeutics, Inc. announced that FDA grants Priority Review for sNDA to convert FILSPARI® (sparsentan) from accelerated approval to full approval for the treatment of IgAN in the U.S
AI Summary
Travere Therapeutics, Inc. announced that the FDA has granted Priority Review for its supplemental New Drug Application (sNDA) to convert FILSPARI® (sparsentan) from accelerated approval to full approval for treating immunoglobulin A nephropathy (IgAN) in the U.S. This decision highlights the FDA's commitment to reviewing the safety and effectiveness data of FILSPARI for patients at risk of rapid disease progression. The review comes with a Prescription Drug User Fee Act (PDUFA) target action date of September 5, 2024, indicating when a decision may be expected. The conversion from accelerated to full approval could enhance the credibility of FILSPARI and potentially broaden its use. This regulatory milestone reflects Travere Therapeutics’ strong commercial progress and increasing patient uptake, reinforcing its focus on advancing treatments for rare diseases and meeting the needs of the IgAN patient community.
Read Announcement
Pegtibatinase (TVT-058) FDA Regulatory Timeline and Events
Pegtibatinase (TVT-058) is a drug developed by Travere Therapeutics for the following indication: Classical homocystinuria (HCU).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Pegtibatinase (TVT-058)
- Announced Date:
- September 3, 2025
- Indication:
- Classical homocystinuria (HCU)
Announcement
Travere Therapeutics, Inc announced that the Company shared two oral presentations in classical homocystinuria (HCU) at the International Congress of Inborn Errors of Metabolism, taking place September 2-6 in Kyoto, Japan.
AI Summary
Travere Therapeutics announced two oral presentations on classical homocystinuria (HCU) at the International Congress of Inborn Errors of Metabolism, September 2–6 in Kyoto, Japan.
The first presentation (OP12-5) on September 3 at 16:00–17:30 JST in Room 3 of the Kyoto International Conference Center will share long-term safety and efficacy data from the Phase 1/2 COMPOSE open-label extension study. At 2.5 mg/kg twice weekly, pegtibatinase reduced total homocysteine by 53.5% and methionine by 67.1% over 50 weeks, with levels remaining below the 100 µM clinical guideline. The therapy was well-tolerated and showed no new safety signals.
The second presentation (OP14-5), also on September 3 at 16:00–17:30 JST in Room 3, will report findings from an open-label, prospective study to determine the optimal HCU treatment in infants identified through newborn screening in Qatar.
Travere remains on track to restart enrollment in the pivotal Phase 3 HARMONY Study evaluating pegtibatinase in 2026.
Read Announcement- Drug:
- Pegtibatinase (TVT-058)
- Announced Date:
- September 26, 2024
- Indication:
- Classical homocystinuria (HCU)
Announcement
Travere Therapeutics, announced a voluntary pause of enrollment in the Phase 3 HARMONY Study evaluating pegtibatinase for the treatment of classical homocystinuria (HCU).
AI Summary
Travere Therapeutics announced a voluntary pause of enrollment in the Phase 3 HARMONY Study for pegtibatinase, a treatment for classical homocystinuria (HCU). The decision was made after the company determined that the desired drug substance profile was not achieved during a recent scale-up process. This pause gives Travere time to make the necessary process improvements in manufacturing to support commercial-scale production and full patient enrollment later.
Patients already enrolled in the study will continue receiving their medication from small-scale batches, which are not affected by the current manufacturing issues. The company is working with its external manufacturing partners to refine the production process, with plans to restart patient enrollment as early as 2026. Travere remains committed to advancing pegtibatinase, aiming to be the first disease-modifying therapy for classical HCU.
Read Announcement- Drug:
- Pegtibatinase (TVT-058)
- Announced Date:
- August 22, 2024
- Indication:
- Classical homocystinuria (HCU)
Announcement
Travere Therapeutics, Inc announced that the Company will present two posters in classical homocystinuria (HCU) at the Society for the Study of Inborn Errors of Metabolism (SSIEM) annual symposium in Porto, Portugal, September 3-6, 2024.
AI Summary
Travere Therapeutics announced that it will present two posters on classical homocystinuria (HCU) at the Society for the Study of Inborn Errors of Metabolism (SSIEM) annual symposium in Porto, Portugal, from September 3-6, 2024. The posters will detail their innovative clinical trial designs for pegtibatinase, an investigational enzyme replacement therapy aimed at addressing classical HCU.
One poster will focus on the designs of the pivotal Phase 3 HARMONY study and the ENSEMBLE long-term extension study that explore the therapy’s potential to replace the deficient CBS enzyme activity. The other poster will present the trial design for cohort 7 of the Phase 1/2 COMPOSE study, which looks into evaluating the safety of pegtibatinase in pediatric patients aged 5 to less than 12 years. Travere’s leadership believes these studies highlight a promising step toward a disease-modifying treatment for people living with classical HCU.
Read Announcement
Sparsentan (DUPLEX) FDA Regulatory Events
Sparsentan (DUPLEX) is a drug developed by Travere Therapeutics for the following indication: Focal Segmental Glomerulosclerosis (FSGS).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Sparsentan (DUPLEX)
- Announced Date:
- March 17, 2025
- Indication:
- Focal Segmental Glomerulosclerosis (FSGS)
Announcement
Travere Therapeutics, Inc., announced the Company has submitted a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) seeking priority review for traditional approval of FILSPARI® (sparsentan) for the treatment of focal segmental glomerulosclerosis (FSGS).
AI Summary
Travere Therapeutics recently submitted a supplemental New Drug Application (sNDA) to the FDA, seeking a priority review for FILSPARI® (sparsentan) for the treatment of focal segmental glomerulosclerosis (FSGS). This application is supported by data from two major clinical studies – the Phase 3 DUPLEX Study and the Phase 2 DUET Study. These studies involved both adults and pediatric patients, marking some of the largest head-to-head trials in FSGS to date.
If approved, FILSPARI could become the first and only FDA-approved treatment for FSGS, offering a new treatment option for patients with this rare kidney disorder that often leads to kidney failure. The company now awaits the FDA’s decision, which will be made after an initial 60-day review period, marking a significant step toward improving outcomes for those affected by FSGS.
Read Announcement
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