Travere Therapeutics Submits sNDA to FDA for Approval of FILSPARI® (sparsentan) for the Treatment of FSGS
Travere Therapeutics (TVTX) has submitted a supplemental New Drug Application (sNDA) to the FDA seeking priority review for FILSPARI® (sparsentan) to treat focal segmental glomerulosclerosis (FSGS). The submission is backed by Phase 3 DUPLEX and Phase 2 DUET studies, which demonstrated FILSPARI's superior efficacy in reducing proteinuria compared to irbesartan in both adults and children.
FILSPARI, a non-immunosuppressive oral medication targeting podocyte injury, could become the first FDA-approved treatment for FSGS, a rare kidney condition and leading cause of kidney failure. The drug is currently approved for IgA nephropathy to slow kidney function decline.
The FDA has 60 days to accept the application for review, with a decision expected in Q2 2025. Additionally, the FDA has deemed REMS monitoring for embryo-fetal toxicity no longer necessary, and the company plans to submit an amendment to modify liver monitoring requirements, with a PDUFA target date of August 28, 2025.
Travere Therapeutics (TVTX) ha presentato una domanda supplementare di registrazione di un nuovo farmaco (sNDA) alla FDA, richiedendo una revisione prioritaria per FILSPARI® (sparsentan) per il trattamento della glomerulosclerosi segmentaria focale (FSGS). La presentazione è supportata dagli studi di Fase 3 DUPLEX e di Fase 2 DUET, che hanno dimostrato l'eccellente efficacia di FILSPARI nella riduzione della proteinuria rispetto all'irbesartan sia negli adulti che nei bambini.
FILSPARI, un farmaco orale non immunosoppressivo mirato a lesioni podocitarie, potrebbe diventare il primo trattamento approvato dalla FDA per la FSGS, una rara condizione renale e principale causa di insufficienza renale. Il farmaco è attualmente approvato per la nefropatia da IgA per rallentare il declino della funzione renale.
La FDA ha 60 giorni per accettare la domanda per la revisione, con una decisione attesa nel secondo trimestre del 2025. Inoltre, la FDA ha ritenuto che il monitoraggio REMS per la tossicità embrio-fetale non sia più necessario, e l'azienda prevede di presentare un emendamento per modificare i requisiti di monitoraggio epatico, con una data obiettivo PDUFA del 28 agosto 2025.
Travere Therapeutics (TVTX) ha presentado una solicitud suplementaria de nuevo medicamento (sNDA) a la FDA solicitando una revisión prioritaria para FILSPARI® (sparsentan) para tratar la glomeruloesclerosis segmentaria focal (FSGS). La presentación está respaldada por los estudios de Fase 3 DUPLEX y de Fase 2 DUET, que demostraron la superior eficacia de FILSPARI en la reducción de proteinuria en comparación con irbesartán tanto en adultos como en niños.
FILSPARI, un medicamento oral no inmunosupresor dirigido a lesiones podocitarias, podría convertirse en el primer tratamiento aprobado por la FDA para la FSGS, una rara condición renal y principal causa de insuficiencia renal. El medicamento está actualmente aprobado para la nefropatía por IgA para ralentizar el deterioro de la función renal.
La FDA tiene 60 días para aceptar la solicitud para revisión, con una decisión esperada en el segundo trimestre de 2025. Además, la FDA ha considerado que la monitorización REMS para la toxicidad embrio-fetal ya no es necesaria, y la empresa planea presentar una enmienda para modificar los requisitos de monitoreo hepático, con una fecha objetivo PDUFA del 28 de agosto de 2025.
Travere Therapeutics (TVTX)는 FILSPARI® (sparsentan)을 초점 분절 사구체 경화증(FSGS) 치료를 위해 FDA에 우선 심사를 요청하는 보충 신약 신청(sNDA)을 제출했습니다. 이 제출은 성인과 어린이 모두에서 irbesartan에 비해 proteinuria 감소에서 FILSPARI의 우수한 효능을 입증한 3상 DUPLEX 및 2상 DUET 연구에 의해 뒷받침됩니다.
FILSPARI는 podocyte 손상을 겨냥한 비면역억제 경구 약물로, FSGS에 대해 FDA에서 승인된 첫 번째 치료제가 될 수 있습니다. 이는 드문 신장 질환이며 신부전의 주요 원인입니다. 이 약물은 현재 IgA 신병증에 대해 신장 기능 저하를 늦추기 위해 승인되었습니다.
FDA는 심사 요청을 수락할 수 있는 60일의 시간을 가지며, 결정은 2025년 2분기에 예상됩니다. 또한, FDA는 태아 독성에 대한 REMS 모니터링이 더 이상 필요하지 않다고 판단했으며, 회사는 간 모니터링 요구 사항을 수정하기 위한 수정안을 제출할 계획이며, PDUFA 목표 날짜는 2025년 8월 28일입니다.
Travere Therapeutics (TVTX) a soumis une demande de médicament nouveau supplémentaire (sNDA) à la FDA pour demander une révision prioritaire de FILSPARI® (sparsentan) afin de traiter la glomérulosclérose segmentaire focale (FSGS). Cette soumission est soutenue par les études de Phase 3 DUPLEX et de Phase 2 DUET, qui ont démontré l'efficacité supérieure de FILSPARI dans la réduction de la protéinurie par rapport à l'irbésartan chez les adultes et les enfants.
FILSPARI, un médicament oral non immunosuppresseur ciblant les lésions podocytaires, pourrait devenir le premier traitement approuvé par la FDA pour la FSGS, une maladie rénale rare et une cause majeure d'insuffisance rénale. Le médicament est actuellement approuvé pour la néphropathie à IgA afin de ralentir le déclin de la fonction rénale.
La FDA dispose de 60 jours pour accepter la demande d'examen, avec une décision attendue au deuxième trimestre 2025. De plus, la FDA a estimé que la surveillance REMS pour la toxicité embryon-fœtale n'est plus nécessaire, et l'entreprise prévoit de soumettre un amendement pour modifier les exigences de surveillance hépatique, avec une date cible PDUFA du 28 août 2025.
Travere Therapeutics (TVTX) hat einen ergänzenden Antrag auf Zulassung eines neuen Arzneimittels (sNDA) bei der FDA eingereicht, in dem eine priorisierte Prüfung für FILSPARI® (sparsentan) zur Behandlung der fokalen segmentalen Glomerulosklerose (FSGS) beantragt wird. Der Antrag wird durch die Phase-3-Studien DUPLEX und Phase-2-Studien DUET unterstützt, die die überlegene Wirksamkeit von FILSPARI bei der Reduktion von Proteinurie im Vergleich zu Irbesartan sowohl bei Erwachsenen als auch bei Kindern gezeigt haben.
FILSPARI, ein nicht immunsuppressives orales Medikament, das auf Podozytenverletzungen abzielt, könnte die erste von der FDA genehmigte Behandlung für FSGS werden, eine seltene Nierenerkrankung und eine der Hauptursachen für Nierenversagen. Das Medikament ist derzeit zur Verlangsamung des Nierenfunktionsverlusts bei IgA-Nephropathie zugelassen.
Die FDA hat 60 Tage Zeit, um den Antrag auf Überprüfung anzunehmen, mit einer Entscheidung, die im 2. Quartal 2025 erwartet wird. Darüber hinaus hat die FDA entschieden, dass das REMS-Monitoring auf embryonale und fetale Toxizität nicht mehr erforderlich ist, und das Unternehmen plant, eine Änderung zur Anpassung der Anforderungen an die Leberüberwachung einzureichen, mit einem PDUFA-Zieldatum vom 28. August 2025.
- Potential to become first FDA-approved treatment for FSGS
- Superior efficacy demonstrated in both Phase 2 and Phase 3 trials
- FDA removes REMS monitoring requirement for embryo-fetal toxicity
- Well-tolerated safety profile consistent across clinical trials
- FDA review timeline uncertain pending application acceptance
- Still requires ongoing liver monitoring under REMS program
Insights
Travere's sNDA submission for FILSPARI represents a significant regulatory milestone with substantial market implications. The company is pursuing priority review status, which could accelerate the standard 10-month review timeline to 6 months if granted.
The FDA's decision to eliminate REMS monitoring for embryo-fetal toxicity is particularly noteworthy - it indicates a growing confidence in the drug's safety profile and reduces the burden on prescribers, potentially enhancing adoption rates. This regulatory easing, combined with the planned simplification of liver monitoring requirements, creates a more favorable prescribing environment that could meaningfully impact uptake if approved for FSGS.
FSGS represents a critical unmet medical need as the leading cause of kidney failure with no FDA-approved treatments. The submission leverages data from both Phase 3 DUPLEX and Phase 2 DUET studies - impressive clinical evidence spanning both adult and pediatric populations. This comprehensive data package strengthens the application considerably.
First-mover advantage in rare diseases typically translates to dominant market position, especially given the non-immunosuppressive mechanism of FILSPARI that differentiates it from current off-label approaches. The dual blockade of endothelin and angiotensin receptors provides a novel approach focusing on podocyte protection rather than symptom management.
FILSPARI's potential approval for FSGS would address one of nephrology's most challenging conditions. FSGS patients face progressive proteinuria leading to kidney function decline with effective interventions. Current approaches rely heavily on off-label ACE inhibitors, ARBs, and immunosuppressants with suboptimal efficacy and significant side effects.
The clinical data demonstrating "rapid, superior and sustained reductions in proteinuria" versus irbesartan (a standard-of-care ARB) is compelling. Proteinuria reduction correlates strongly with preserved kidney function, making this endpoint clinically meaningful. The dual mechanism targeting both endothelin and angiotensin pathways addresses the complex pathophysiology of podocyte injury in FSGS more comprehensively than single-pathway approaches.
FILSPARI's established safety profile in IgA nephropathy provides reassurance regarding its tolerability. The non-immunosuppressive approach is particularly valuable given the adverse event profile and variable responses to immunosuppressants currently used off-label.
The potential approval would transform FSGS treatment protocols nationally, offering nephrologists the first evidence-based therapy specifically validated in this population. For patients with this devastating condition, FILSPARI represents the first purpose-designed therapy targeting underlying disease mechanisms rather than symptomatic management. This would represent a paradigm shift in FSGS treatment approach.
sNDA submission based on results from Phase 3 DUPLEX and Phase 2 DUET studies of FILSPARI in FSGS
If approved, FILSPARI could become the first and only FDA-approved treatment for FSGS, a rare kidney condition and a leading cause of kidney failure
Additionally, the FDA notified the Company that REMS monitoring for embryo-fetal toxicity is no longer necessary; the Company plans to submit an amendment to the REMS sNDA currently under review for modification of liver monitoring
SAN DIEGO, March 17, 2025 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc., (Nasdaq: TVTX) today 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). The submission is supported by results from the Phase 3 DUPLEX Study and the Phase 2 DUET Study, two of the largest head-to-head interventional studies conducted to date in adult and pediatric patients with FSGS.
“There is a profound and urgent need for effective treatment options that can target glomerular injury, reduce proteinuria, and preserve kidney function in FSGS,” said Eric Dube, Ph.D., president and chief executive officer of Travere Therapeutics. “Since its approval in IgA nephropathy, we have seen the positive impact FILSPARI can have on patients living with rare kidney disease. We have great hope to potentially bring FILSPARI as the first approved treatment for patients with FSGS and this sNDA submission is an important next step toward that goal. We look forward to the upcoming review process.”
FILSPARI is a non-immunosuppressive, oral medication that directly targets podocyte injury by selectively blocking the endothelin A receptor (ETAR) and the angiotensin II subtype 1 receptor (AT1R). It is currently approved to slow kidney function decline in adults with IgA nephropathy, a leading cause of kidney failure. The DUPLEX and DUET studies demonstrated that FILSPARI provided rapid, superior and sustained reductions in proteinuria when compared with maximum labeled dose irbesartan, in children and adults with FSGS. In the DUPLEX and DUET studies, FILSPARI was well-tolerated with a safety profile that was consistent across all clinical trials conducted to date.
The FDA has 60 days from the receipt of the application to determine whether to accept it for review. The Company expects to receive notice regarding the acceptance for review of the sNDA submission as well as the timeline for sNDA review from the FDA in the second quarter of 2025.
Additionally, the FDA recently notified the Company that the REMS is no longer necessary to ensure the benefits of FILSPARI outweigh the risk of embryo-fetal toxicity and to minimize the burden on the healthcare delivery system. The Company plans to submit a REMS modification to remove the need to monitor the risk of embryo-fetal toxicity as an amendment to the REMS sNDA currently under review for potential modification of liver monitoring. The FDA indicated that this amendment is not expected to impact the review timeline and the Company continues to expect a REMS modification PDUFA target action date of August 28, 2025.
About FSGS
Focal segmental glomerulosclerosis (FSGS) is a rare proteinuric kidney disorder in both children and adults that is estimated to affect more than 40,000 patients in the U.S. with similar prevalence in Europe. The disorder is defined by progressive scarring of the kidney and often leads to kidney failure. FSGS is characterized by proteinuria, where protein leaks into the urine due to a breakdown of the normal filtration mechanism in the kidney. Once in the urine, protein is considered to be toxic to other parts of the kidney, especially the tubules, and is believed to contribute to further disease progression. Other common symptoms include swelling in parts of the body, known as edema, as well as low blood albumin levels, abnormal lipid profiles and hypertension. There are currently no FDA-approved pharmacologic therapies for FSGS.
About the DUPLEX and DUET Studies
The Phase 3 DUPLEX Study is the largest interventional study to date in FSGS, and the only study in FSGS against a maximally dosed active comparator. While DUPLEX achieved its pre-specified interim FSGS partial remission of proteinuria (FPRE) endpoint with statistical significance at 36 weeks, it did not achieve the primary efficacy eGFR slope endpoint over 108 weeks of treatment. The two-year results from the study were published in the New England Journal of Medicine and showed that sparsentan delivered clinically meaningful benefit at 108 weeks with significant proteinuria reduction, higher rates of partial and complete remission, and a lower rate of end-stage kidney disease compared to the active control. The Phase 2 DUET Study of sparsentan in FSGS met the primary efficacy endpoint for the combined treatment group, demonstrating a greater than two-fold reduction in proteinuria compared to irbesartan. Sparsentan was well-tolerated with a safety profile that was consistent across all clinical trials conducted to date and comparable to the active control, irbesartan, including no drug-induced liver injury and no fluid overload. Patients who completed the DUPLEX and DUET double-blind portions of the studies on treatment were eligible to participate in the open-label extension of the trials.
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
FILSPARI® (sparsentan) U.S. Indication
FILSPARI (sparsentan) is indicated to slow kidney function decline in adults with primary immunoglobulin A nephropathy (IgAN) who are at risk for disease progression.
IMPORTANT SAFETY INFORMATION
BOXED WARNING: HEPATOTOXICITY AND EMBRYO-FETAL TOXICITY
Because of the risks of hepatotoxicity and birth defects, FILSPARI is available only through a restricted program called the FILSPARI REMS. Under the FILSPARI REMS, prescribers, patients and pharmacies must enroll in the program.
Hepatotoxicity
Some Endothelin Receptor Antagonists (ERAs) have caused elevations of aminotransferases, hepatotoxicity, and liver failure. In clinical studies, elevations in aminotransferases (ALT or AST) of at least 3-times the Upper Limit of Normal (ULN) have been observed in up to
Measure transaminases and bilirubin before initiating treatment and monthly for the first 12 months, and then every 3 months during treatment. Interrupt treatment and closely monitor patients who develop aminotransferase elevations more than 3x ULN.
FILSPARI should generally be avoided in patients with elevated aminotransferases (>3x ULN) at baseline because monitoring for hepatotoxicity may be more difficult and these patients may be at increased risk for serious hepatotoxicity.
Embryo-Fetal Toxicity
FILSPARI can cause major birth defects if used by pregnant patients based on animal data. Therefore, pregnancy testing is required before the initiation of treatment, during treatment and one month after discontinuation of treatment with FILSPARI. Patients who can become pregnant must use effective contraception before the initiation of treatment, during treatment, and for one month after discontinuation of treatment with FILSPARI.
Contraindications
FILSPARI is contraindicated in patients who are pregnant. Do not coadminister FILSPARI with angiotensin receptor blockers (ARBs), ERAs, or aliskiren.
Warnings and Precautions
- Hepatotoxicity: Elevations in ALT or AST of at least 3-fold ULN have been observed in up to
3.5% of FILSPARI-treated patients, including cases confirmed with rechallenge. While no concurrent elevations in bilirubin >2-times ULN or cases of liver failure were observed in FILSPARI-treated patients, some ERAs have caused elevations of aminotransferases, hepatotoxicity, and liver failure. To reduce the risk of potential serious hepatotoxicity, measure serum aminotransferase levels and total bilirubin prior to initiation of treatment and monthly for the first 12 months, then every 3 months during treatment.
Advise patients with symptoms suggesting hepatotoxicity (nausea, vomiting, right upper quadrant pain, fatigue, anorexia, jaundice, dark urine, fever, or itching) to immediately stop treatment with FILSPARI and seek medical attention. If aminotransferase levels are abnormal at any time during treatment, interrupt FILSPARI and monitor as recommended.
Consider re-initiation of FILSPARI only when hepatic enzyme levels and bilirubin return to pretreatment values and only in patients who have not experienced clinical symptoms of hepatotoxicity. Avoid initiation of FILSPARI in patients with elevated aminotransferases (>3x ULN) prior to drug initiation because monitoring hepatotoxicity in these patients may be more difficult and these patients may be at increased risk for serious hepatotoxicity.
- Embryo-Fetal Toxicity: FILSPARI can cause fetal harm when administered to a pregnant patient and is contraindicated during pregnancy. Advise patients who can become pregnant of the potential risk to a fetus. Obtain a pregnancy test prior to initiation of treatment with FILSPARI, monthly during treatment, and one month after discontinuation of treatment. Advise patients who can become pregnant to use effective contraception prior to initiation of treatment, during treatment, and for one month after discontinuation of treatment with FILSPARI.
- FILSPARI REMS: Due to the risk of hepatotoxicity and embryo-fetal toxicity, FILSPARI is available only through a restricted program called the FILSPARI REMS. Prescribers, patients, and pharmacies must be enrolled in the REMS program and comply with all requirements (www.filsparirems.com).
- Hypotension: Hypotension has been observed in patients treated with ARBs and ERAs. There was a greater incidence of hypotension-associated adverse events, some serious, including dizziness, in patients treated with FILSPARI compared to irbesartan. In patients at risk for hypotension, consider eliminating or adjusting other antihypertensive medications and maintaining appropriate volume status. If hypotension develops, despite elimination or reduction of other antihypertensive medications, consider a dose reduction or dose interruption of FILSPARI. A transient hypotensive response is not a contraindication to further dosing of FILSPARI, which can be given once blood pressure has stabilized.
- Acute Kidney Injury: Monitor kidney function periodically. Drugs that inhibit the renin-angiotensin system (RAS) can cause kidney injury. Patients whose kidney function may depend in part on the activity of the RAS (e.g., patients with renal artery stenosis, chronic kidney disease, severe congestive heart failure, or volume depletion) may be at particular risk of developing acute kidney injury on FILSPARI. Consider withholding or discontinuing therapy in patients who develop a clinically significant decrease in kidney function while on FILSPARI.
- Hyperkalemia: Monitor serum potassium periodically and treat appropriately. Patients with advanced kidney disease, taking concomitant potassium-increasing drugs (e.g., potassium supplements, potassium-sparing diuretics), or using potassium-containing salt substitutes are at increased risk for developing hyperkalemia. Dosage reduction or discontinuation of FILSPARI may be required.
- Fluid Retention: Fluid retention may occur with ERAs, and has been observed in clinical studies with FILSPARI. FILSPARI has not been evaluated in patients with heart failure. If clinically significant fluid retention develops, evaluate the patient to determine the cause and the potential need to initiate or modify the dose of diuretic treatment then consider modifying the dose of FILSPARI.
Most common adverse reactions
The most common adverse reactions (≥
Drug interactions
- Renin-Angiotensin System (RAS) Inhibitors and ERAs: Do not coadminister FILSPARI with ARBs, ERAs, or aliskiren due to increased risks of hypotension, syncope, hyperkalemia, and changes in renal function (including acute renal failure).
- Strong and Moderate CYP3A Inhibitors: Avoid concomitant use of FILSPARI with strong CYP3A inhibitors. If a strong CYP3A inhibitor cannot be avoided, interrupt FILSPARI treatment. When resuming treatment with FILSPARI, consider dose titration. Monitor blood pressure, serum potassium, edema, and kidney function regularly when used concomitantly with moderate CYP3A inhibitors. Concomitant use with a strong CYP3A inhibitor increases sparsentan exposure which may increase the risk of FILSPARI adverse reactions.
- Strong CYP3A Inducers: Avoid concomitant use with a strong CYP3A inducer. Concomitant use with a strong CYP3A inducer decreases sparsentan exposure which may reduce FILSPARI efficacy.
- Antacids and Acid Reducing Agents: Administer FILSPARI 2 hours before or after administration of antacids. Avoid concomitant use of acid reducing agents (histamine H2 receptor antagonist and PPI proton pump inhibitor) with FILSPARI. Sparsentan exhibits pH-dependent solubility. Antacids or acid reducing agents may decrease sparsentan exposure which may reduce FILSPARI efficacy.
- Non-Steroidal Anti-Inflammatory Agents (NSAIDs), Including Selective Cyclooxygenase-2 (COX-2) Inhibitors: Monitor for signs of worsening renal function with concomitant use with NSAIDs (including selective COX-2 inhibitors). In patients with volume depletion (including those on diuretic therapy) or with impaired kidney function, concomitant use of NSAIDs (including selective COX-2 inhibitors) with drugs that antagonize the angiotensin II receptor may result in deterioration of kidney function, including possible kidney failure.
- CYP2B6, 2C9, and 2C19 Substrates: Monitor for efficacy of concurrently administered CYP2B6, 2C9, and 2C19 substrates and consider dosage adjustment in accordance with the Prescribing Information. Sparsentan decreases exposure of these substrates, which may reduce efficacy related to these substrates.
- P-gp and BCRP Substrates: Avoid concomitant use of sensitive substrates of P-gp and BCRP with FILSPARI. Sparsentan may increase exposure of these transporter substrates, which may increase the risk of adverse reactions related to these substrates.
- Agents Increasing Serum Potassium: Monitor serum potassium frequently in patients treated with FILSPARI and other agents that increase serum potassium. Concomitant use of FILSPARI with potassium-sparing diuretics, potassium supplements, potassium-containing salt substitutes, or other drugs that raise serum potassium levels may result in hyperkalemia.
Please see the full Prescribing Information, including BOXED WARNING, for additional Important Safety Information.
Forward Looking Statements
This press release contains “forward-looking statements” as that term is defined in the Private Securities Litigation Reform Act of 1995. Without limiting the foregoing, these statements are often identified by the words “on-track,” “positioned,” “look forward to,” “will,” “would,” “may,” “might,” “believes,” “anticipates,” “plans,” “expects,” “intends,” “potential,” or similar expressions. In addition, expressions of strategies, intentions or plans are also forward-looking statements. Such forward-looking statements include, but are not limited to, references to: statements and expectations regarding the Company’s sNDA for FILSPARI in FSGS, including expectations regarding the timing and outcome thereof, the potential for the FDA to accept the filing and grant priority review, and related matters; statements regarding the potential for FILSPARI to become the first and only FDA-approved medicine indicated for FSGS; statements and expectations regarding potential changes to the REMS, including the Company’s plans to submit an amendment to the REMS sNDA currently under review for potential modification of liver monitoring and the expected timing and outcome thereof; and references to the estimated size of the patient population. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could delay, divert or change any of them, and could cause actual outcomes and results to differ materially from current expectations. No forward-looking statement can be guaranteed. Among the factors that could cause actual results to differ materially from those indicated in the forward-looking statements are risks and uncertainties related to the Company’s sNDA for FILSPARI in FSGS, including the timing and outcome thereof. There is no guarantee that the FDA will accept the sNDA for filing, grant priority review of the sNDA or grant approval of FILSPARI for FSGS on the anticipated timeline, or at all. The Company also faces risks and uncertainties related to the Company’s planned submission of an amendment to the REMS sNDA currently under review for potential modification of liver monitoring, and the anticipated timing and outcome thereof, risks related to its business and finances in general, the success of its commercial products, risks and uncertainties associated with its preclinical and clinical stage pipeline, risks and uncertainties associated with the regulatory review and approval process, risks and uncertainties associated with enrollment of clinical trials for rare diseases, and risks that ongoing or planned clinical trials may not succeed or may be delayed for safety, regulatory or other reasons. Specifically, the Company faces risks associated with the ongoing commercial launch of FILSPARI in IgAN, the timing and potential outcome of its and its partners’ clinical studies, market acceptance of its commercial products including efficacy, safety, price, reimbursement, and benefit over competing therapies, risks related to the challenges of manufacturing scale-up, risks associated with the successful development and execution of commercial strategies for such products, including FILSPARI, and risks and uncertainties related to the new administration and matters related to the funding and staffing of government agencies including the FDA. The Company also faces the risk that it will be unable to raise additional funding that may be required to complete development of any or all of its product candidates, including as a result of macroeconomic conditions; risks relating to the Company’s dependence on contractors for clinical drug supply and commercial manufacturing; uncertainties relating to patent protection and exclusivity periods and intellectual property rights of third parties; risks associated with regulatory interactions; and risks and uncertainties relating to competitive products, including current and potential future generic competition with certain of the Company’s products, and technological changes that may limit demand for the Company’s products. The Company also faces additional risks associated with global and macroeconomic conditions, including health epidemics and pandemics, including risks related to potential disruptions to clinical trials, commercialization activity, supply chain, and manufacturing operations. You are cautioned not to place undue reliance on these forward-looking statements as there are important factors that could cause actual results to differ materially from those in forward-looking statements, many of which are beyond our control. The Company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise. Investors are referred to the full discussion of risks and uncertainties, including under the heading “Risk Factors”, as included in the Company’s most recent Form 10-K, Form 10-Q and other filings with the Securities and Exchange Commission.
Contact Info
| Media: 888-969-7879 mediarelations@travere.com | Investors: 888-969-7879 IR@travere.com |
FAQ
What clinical trial results support FILSPARI's sNDA submission for FSGS treatment?
When will the FDA decide on TVTX's FILSPARI sNDA acceptance for FSGS?
What changes are being made to FILSPARI's REMS monitoring requirements?
How does FILSPARI work in treating FSGS patients?
What is the current FDA-approved indication for FILSPARI (TVTX)?
