New Drug Application initiated with U.S. FDA for TAR-200, the first and only intravesical drug releasing system for patients with BCG-unresponsive high-risk non-muscle-invasive bladder cancer
Johnson & Johnson (NYSE: JNJ) has initiated a New Drug Application with the FDA for TAR-200, the first intravesical drug releasing system for BCG-unresponsive high-risk non-muscle-invasive bladder cancer (HR-NMIBC). The application is being reviewed under the FDA's Real-Time Oncology Review program, based on impressive Phase 2b SunRISe-1 study results showing an 83.5% complete response rate.
The study demonstrated strong durability, with 82% of responders maintaining response at a median follow-up of nine months. Safety data revealed low occurrence (9%) of Grade 3 or higher treatment-related adverse events, with only 6% of patients discontinuing due to adverse events and no treatment-related deaths reported.
TAR-200 delivers gemcitabine directly into the bladder through a quick outpatient procedure requiring no anesthesia. The FDA granted Breakthrough Therapy Designation to TAR-200 in December 2023 for treating adult patients with BCG-unresponsive HR-NMIBC with CIS who are ineligible for or have declined radical cystectomy.
Johnson & Johnson (NYSE: JNJ) ha avviato una Richiesta di Nuovo Farmaco presso la FDA per TAR-200, il primo sistema di rilascio intravescicale di farmaci per il cancro della vescica non muscolare invasivo ad alto rischio (HR-NMIBC) non reattivo al BCG. La richiesta è attualmente in fase di revisione nel contesto del programma di Revisione Oncologica in Tempo Reale della FDA, basata su risultati impressionanti dello studio di Fase 2b SunRISe-1, che mostrano un tasso di risposta completa dell'83,5%.
Lo studio ha dimostrato una forte durabilità, con l'82% dei rispondenti che ha mantenuto la risposta a un follow-up mediano di nove mesi. I dati di sicurezza hanno rivelato una bassa incidenza (9%) di eventi avversi correlati al trattamento di Grado 3 o superiore, con solo il 6% dei pazienti che ha interrotto il trattamento a causa di eventi avversi e nessun decesso correlato al trattamento segnalato.
TAR-200 somministra gemcitabina direttamente nella vescica attraverso una rapida procedura ambulatoriale che non richiede anestesia. La FDA ha concesso la Designazione di Terapia Avanzata a TAR-200 nel dicembre 2023 per il trattamento di pazienti adulti con HR-NMIBC non reattivo al BCG con CIS che non sono idonei o che hanno rifiutato la cistectomia radicale.
Johnson & Johnson (NYSE: JNJ) ha iniciado una Solicitud de Nuevo Medicamento con la FDA para TAR-200, el primer sistema de liberación intravesical de fármacos para el cáncer de vejiga no invasivo de alto riesgo (HR-NMIBC) que no responde al BCG. La solicitud está siendo revisada bajo el programa de Revisión Oncológica en Tiempo Real de la FDA, basado en resultados impresionantes del estudio de Fase 2b SunRISe-1 que muestran un tasa de respuesta completa del 83.5%.
El estudio demostró una fuerte durabilidad, con el 82% de los respondedores manteniendo la respuesta a un seguimiento medio de nueve meses. Los datos de seguridad revelaron una baja ocurrencia (9%) de eventos adversos relacionados con el tratamiento de Grado 3 o superior, con solo el 6% de los pacientes interrumpiendo el tratamiento debido a eventos adversos y sin muertes relacionadas con el tratamiento reportadas.
TAR-200 entrega gemcitabina directamente en la vejiga a través de un procedimiento ambulatorio rápido que no requiere anestesia. La FDA otorgó la Designación de Terapia Innovadora a TAR-200 en diciembre de 2023 para el tratamiento de pacientes adultos con HR-NMIBC no responsive al BCG con CIS que no son elegibles o que han rechazado la cistectomía radical.
존슨 & 존슨 (NYSE: JNJ)는 FDA에 TAR-200에 대한 새로운 약물 신청을 시작했습니다. 이는 BCG에 반응하지 않는 고위험 비근육 침습성 방광암(HR-NMIBC)을 위한 첫 번째 방광 내 약물 방출 시스템입니다. 이 신청은 FDA의 실시간 종양학 검토 프로그램에 따라 검토되고 있으며, 인상적인 2b 단계의 SunRISe-1 연구 결과를 바탕으로 83.5%의 완전 반응률을 보여줍니다.
이번 연구는 강한 내구성을 입증했으며, 응답자의 82%가 9개월의 중간 추적 조사에서 반응을 유지했습니다. 안전성 데이터는 Grade 3 이상의 치료 관련 불리한 사건이 9%로 낮게 발생했다고 밝혔으며, 환자의 6%만이 불리한 사건으로 치료를 중단했고 치료 관련 사망 사례는 보고되지 않았습니다.
TAR-200은 마취 없이 빠른 외래 절차를 통해 방광에 직접 젬시타빈을 전달합니다. FDA는 2023년 12월에 CIS와 함께 BCG에 반응하지 않는 HR-NMIBC 치료를 위한 TAR-200의 혁신 치료 지정(Breakthrough Therapy Designation)을 부여했습니다.
Johnson & Johnson (NYSE: JNJ) a lancé une Demande de Nouveau Médicament auprès de la FDA pour TAR-200, le premier système de libération intravésicale de médicament pour le cancer de la vessie non invasif à risque élevé (HR-NMIBC) non réactif au BCG. La demande est en cours d'examen dans le cadre du programme d'examen oncologique en temps réel de la FDA, sur la base des résultats impressionnants de l'étude de Phase 2b SunRISe-1 montrant un taux de réponse complet de 83.5%.
L'étude a démontré une forte durabilité, avec 82% des répondants maintenant leur réponse lors d'un suivi médian de neuf mois. Les données de sécurité ont révélé une faible occurrence (9%) d'événements indésirables liés au traitement de Grade 3 ou supérieur, avec seulement 6% des patients interrompant le traitement en raison d'événements indésirables et aucun décès lié au traitement signalé.
TAR-200 délivre de la Gemcitabine directement dans la vessie par le biais d'une procédure ambulatoire rapide ne nécessitant pas d'anesthésie. La FDA a accordé à TAR-200 la désignation de thérapie innovante en décembre 2023 pour le traitement des patients adultes atteints de HR-NMIBC non réactif au BCG avec CIS qui ne sont pas éligibles ou qui ont refusé une cystectomie radicale.
Johnson & Johnson (NYSE: JNJ) hat einen Antrag auf neue Arzneimittel bei der FDA für TAR-200 eingereicht, das erste intravesikale Medikamentenfreisetzungssystem für BCG-unempfindlichen, hochriskanten nicht-muskelinvasiven Blasenkrebs (HR-NMIBC). Der Antrag wird im Rahmen des Real-Time Oncology Review-Programms der FDA überprüft, basierend auf beeindruckenden Ergebnissen der Phase 2b SunRISe-1-Studie, die eine vollständige Ansprechrate von 83,5% zeigen.
Die Studie zeigte eine starke Haltbarkeit, wobei 82% der Behandler ihre Reaktion über eine mediane Nachbeobachtungszeit von neun Monaten beibehielten. Die Sicherheitsdaten zeigten eine geringe Häufigkeit (9%) von behandlungsbezogenen unerwünschten Ereignissen der Grad 3 oder höher, wobei nur 6% der Patienten aufgrund unerwünschter Ereignisse die Behandlung abbrachen und keine behandlungsbezogenen Todesfälle gemeldet wurden.
TAR-200 liefert Gemcitabin direkt in die Blase durch ein schnelles ambulatorisches Verfahren, das keine Anästhesie erfordert. Die FDA verlieh TAR-200 im Dezember 2023 den Status einer Durchbruchtherapie zur Behandlung von erwachsenen Patienten mit BCG-unempfindlichem HR-NMIBC mit CIS, die für eine radikale Zystektomie nicht in Frage kommen oder diese abgelehnt haben.
- Achieved 83.5% complete response rate in Phase 2b trial
- 82% of responders maintained response at 9-month median follow-up
- Received FDA Breakthrough Therapy Designation
- Low adverse event profile with only 9% Grade 3 or higher events
- Simple outpatient procedure requiring no anesthesia
- 6% of patients discontinued treatment due to adverse events
Insights
The FDA submission for TAR-200 represents a potential game-changing advancement in bladder cancer treatment. The 83.5% complete response rate from the Phase 2b trial is remarkably high for this indication, substantially exceeding historical benchmarks for BCG-unresponsive NMIBC treatments. The 82% durability rate at 9 months without reinduction is particularly impressive, suggesting superior sustained efficacy compared to current standards.
The safety profile is notably robust, with only
The intravesical drug delivery system represents significant innovation in medical device technology. The system's ability to provide sustained local delivery of gemcitabine directly to the bladder tissue, combined with its rapid outpatient administration protocol, addresses key clinical challenges in bladder cancer treatment. The co-packaged urinary placement catheter design suggests careful consideration of both efficacy and practical implementation in clinical settings.
The Breakthrough Therapy Designation from the FDA, coupled with acceptance into the Real-Time Oncology Review program, indicates strong regulatory confidence in both the technology and its potential impact. This expedited review pathway typically reduces approval timelines by 3-6 months, potentially accelerating market access.
This development could significantly impact JNJ's oncology portfolio and market position. The bladder cancer treatment market is projected to exceed
The innovative combination of drug delivery and medical device technology leverages JNJ's cross-divisional strengths, potentially creating high barriers to competition. Given the strong clinical data and significant unmet need in BCG-unresponsive NMIBC, TAR-200 could achieve peak annual sales of
Application accepted for
"Upon approval, TAR-200 promises to be a meaningful additional treatment option for certain patients with NMIBC, addressing a critical need for people who have had relatively limited therapeutic alternatives. Many patients face life-altering surgical options such as radical cystectomy, which is complete bladder removal," said Yusri Elsayed, M.D., M.H.Sc., Ph.D., Global Therapeutic Head, Oncology, Johnson & Johnson Innovative Medicine. "By combining our expertise in innovative medicine and medical devices, Johnson & Johnson is uniquely positioned to transform how we treat certain types of bladder cancer through the first and only intravesical drug releasing system for this disease. We look forward to working with the FDA in review of this application."
The submission of this innovative intravesical drug releasing system is supported by data from the Phase 2b SunRISe-1 registration study. Data collected through the second quarter of 2024 and presented at the European Society for Medical Oncology (ESMO) 2024 Congress as a late-breaking oral presentation showed an 83.5 percent complete response (CR) rate and highly durable CRs without the need for reinduction – at a median follow-up of nine months, 82 percent of responders maintained response. At data cutoff in May 2024, safety and tolerability data presented at ESMO demonstrated a low occurrence of Grade 3 or higher treatment-related adverse events (TRAEs) (9 percent); five patients had TRAEs leading to discontinuation (6 percent) and no treatment-related deaths were reported.1
TAR-200 is an investigational intravesical drug releasing system designed to provide sustained local delivery of gemcitabine into the bladder. It is placed into the bladder by a healthcare professional using a co-packaged urinary placement catheter in an outpatient setting in under five minutes and without the need for anesthesia.
In December 2023, the FDA granted Breakthrough Therapy Designation (BTD) to TAR-200 for the treatment of adult patients with BCG-unresponsive HR-NMIBC with CIS who are ineligible for or have elected not to undergo radical cystectomy.
About SunRISe-1, Cohort 2
SunRISe-1 (NCT04640623), Cohort 2, is a randomized, parallel-assignment, open-label Phase 2b clinical study evaluating the safety and efficacy of TAR-200 monotherapy alone for BCG-unresponsive HR-NMIBC carcinoma in situ (CIS) patients who are ineligible for, or elected not to undergo, radical cystectomy. The primary endpoint for Cohort 2 is CR rate at any time point. Secondary endpoints include duration of response (DOR), overall survival (OS), pharmacokinetics, quality of life, safety and tolerability.
About High-Risk Non-Muscle-Invasive Bladder Cancer
High-risk non-muscle-invasive bladder cancer (HR-NMIBC) is a type of non-invasive bladder cancer that is more likely to recur or spread beyond the lining of the bladder, called the urothelium, and progress to invasive bladder cancer compared to low-risk NMIBC.2, 3 HR-NMIBC makes up 15-44 percent of patients with NMIBC and is characterized by a high-grade, large tumor size, presence of multiple tumors, and CIS. Radical cystectomy is currently recommended for NMIBC patients who fail BCG therapy, with over 90 percent cancer-specific survival if performed before muscle-invasive progression.4, 5 Given that NMIBC typically affects older patients, many may be unwilling or unfit to undergo radical cystectomy.6 The high rates of recurrence and progression can pose significant morbidity and distress for these patients.2, 5
About Johnson & Johnson
At Johnson & Johnson, we believe health is everything. Our strength in healthcare innovation empowers us to build a world where complex diseases are prevented, treated, and cured, where treatments are smarter and less invasive, and solutions are personal. Through our expertise in Innovative Medicine and MedTech, we are uniquely positioned to innovate across the full spectrum of healthcare solutions today to deliver the breakthroughs of tomorrow and profoundly impact health for humanity. Learn more at https://www.jnj.com or at www.innovativemedicine.jnj.com. Follow us at @JNJInnovMed. Janssen Research & Development, LLC, and Janssen Biotech, Inc., are Johnson & Johnson companies.
Cautions Concerning Forward-Looking Statements
This press release contains "forward-looking statements" as defined in the Private Securities Litigation Reform Act of 1995 regarding product development and the potential benefits and treatment impact of TAR-200. The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could vary materially from the expectations and projections of Janssen Research & Development, LLC, Janssen Biotech, Inc., Janssen Global Services, LLC and/or Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and of obtaining regulatory approvals; uncertainty of commercial success; manufacturing difficulties and delays; competition, including technological advances, new products and patents attained by competitors; challenges to patents; product efficacy or safety concerns resulting in product recalls or regulatory action; changes in behavior and spending patterns of purchasers of healthcare products and services; changes to applicable laws and regulations, including global healthcare reforms; and trends toward healthcare cost containment. A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson's Annual Report on Form 10-K for the fiscal year ended December 31, 2023, including in the sections captioned "Cautionary Note Regarding Forward-Looking Statements" and "Item 1A. Risk Factors," and in Johnson & Johnson's subsequent Quarterly Reports on Form 10-Q and other filings with the Securities and Exchange Commission. Copies of these filings are available online at www.sec.gov, www.jnj.com or on request from Johnson & Johnson. None of Janssen Research & Development, LLC, Janssen Biotech, Inc., Janssen Global Services, LLC nor Johnson & Johnson undertakes to update any forward-looking statement as a result of new information or future events or developments.
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1 Van der Heijden M, et al. TAR-200 +/- Cetrelimab and Cetrelimab Alone in Patients With Bacillus Calmette-Guérin–Unresponsive High-Risk Non–Muscle-Invasive Bladder Cancer: Updated Results From SunRISe-1. ESMO 2024. September 15, 2024. |
2 Grab-Heyne K, Henne C, Mariappan P, et al. Intermediate and high-risk non–muscle-invasive bladder cancer: an overview of epidemiology, burden, and unmet needs. Front Oncol. 2023;13:1170124. |
3 Lieblich A, Henne C, Mariappan P, Geiges G, Pöhlmann J, Pollock RF. The management of non–muscle-invasive bladder cancer: a comparison of European and |
4 Brooks NA, O'Donnell MA. Treatment options in non–muscle-invasive bladder cancer after BCG failure. Indian J Urol. 2015;31(4):312-319. doi:10.4103/0970-1591.166475 |
5 Guancial EA, Roussel B, Bergsma DP, et al. Bladder cancer in the elderly patient: challenges and solutions. Clin Interv Aging. 2015;10:939-949. |
6 Chamie K, Litwin MS, Bassett JC, et al. Recurrence of high-risk bladder cancer: A population-based analysis. Cancer. 2013;119(17):3219-3227. |
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