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 Johnson & Johnson (J&J) announced that the US Food and Drug Administration (FDA) granted Priority Review to the New Drug Application (NDA) filed for TAR-200, an intravesical gemcitabine releasing system, for the treatment of patients with Bacillus Calmette-Guérin (BCG)-unresponsive high-risk non-muscle invasive bladder cancer (HR-NMIBC) with carcinoma in situ (CIS), with or without papillary tumours.
"TAR-200 represents an innovation in drug delivery that has not been seen in decades," said Yusri Elsayed, M.D., M.H.Sc., Ph.D., global therapeutic area head, oncology, Johnson & Johnson Innovative Medicine. "The FDA Priority Review for TAR-200 underscores our mission to fundamentally change the way urologists treat certain types of bladder cancer."
The regulatory submission is supported by data from the phase 2b SunRISe-1 study, which demonstrated an 82.4 per cent complete response (CR) rate with 52.9 per cent of patients remaining cancer-free at least one year or more after achievement of a CR (95 per cent confidence interval [CI], 72.6-89.8). The majority of adverse reactions were mild and moderate. The most common adverse reactions (=10 percent) included pollakiuria, dysuria, urinary tract infection, micturition urgency, hematuria, cystitis noninfective, and urinary tract pain. No systemic adverse reactions were reported. The findings were presented during a plenary session at the April 2025 American Urological Association Annual Meeting.
Despite advancements, there has been little change in the standard of care for patients with HR-NMIBC for over 40 years, and patients have limited treatment options if initial BCG therapy does not work. TAR-200 is the first and only intravesical drug releasing system (iDRS) designed to provide sustained local delivery of a cancer treatment into the bladder. TAR-200 remains in the bladder for three weeks per treatment cycle. A healthcare professional places it into the bladder using a co-packaged urinary placement catheter in an outpatient setting in less than five minutes. There is no need for general anaesthesia, further monitoring, or other restrictions immediately post-insertion within the healthcare provider's office.
TAR-200 is an investigational intravesical gemcitabine releasing system. In January 2025, Johnson & Johnson announced the initiation of a new drug application with the FDA for TAR-200 under the Real-Time Oncology Review (RTOR) programme. 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. The safety and efficacy of TAR-200 are being evaluated in phase 2 and phase 3 studies in patients with NMIBC in SunRISe-1, SunRISe-3 and SunRISe-5 and muscle-invasive bladder cancer (MIBC) in SunRISe-4.
SunRISe-1 (NCT04640623), Cohort 2, is a single arm, open-label phase 2b clinical study evaluating the safety and efficacy of TAR-200 monotherapy for BCG-unresponsive HR-NMIBC patients with carcinoma in situ (CIS) with or without papillary tumours who are ineligible for, or elected not to undergo, radical cystectomy. The primary endpoint for Cohort 2 is complete response (CR) rate at any time point, and secondary endpoints include duration of response (DOR), safety, overall survival and quality of life.
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 muscle invasive bladder cancer compared to low-risk NMIBC. HR-NMIBC is characterized by a high-grade, large tumour size, presence of multiple tumours, and carcinoma in situ (CIS). HR-NMIBC with CIS makes up approximately 10 per cent of patients with NMIBC. Bacillus Calmette-Guérin (BCG), is an intravesical medication administered directly into the bladder that patients have to hold for a few hours. BCG is a weakened form of the bacteria found in tuberculosis treatment, and though effective, some patients become unresponsive to it and experience tolerability challenges. Radical cystectomy is currently recommended for HR-NMIBC patients who fail BCG therapy; it is a life-altering surgery with a high degree of morbidity and impact on life, and has a post-surgery mortality rate of three to eight per cent. Given that HR-NMIBC typically affects older patients, many may be unwilling or unfit to undergo radical cystectomy.
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