FDA Grants Priority Review to Johnson & Johnson's TAR-200 NDA for High-Risk Bladder Cancer

Johnson & Johnson recently announced that the U.S. Food and Drug Administration (FDA) has awarded Priority Review status to its New Drug Application for TAR-200. This innovative drug delivery system, which releases gemcitabine directly into the bladder, is intended for patients who have Bacillus Calmette-Guérin (BCG)-unresponsive high-risk non-muscle invasive bladder cancer (HR-NMIBC) with carcinoma in situ, with or without papillary tumors.

Dr. Yusri Elsayed, Global Therapeutic Area Head for Oncology at Johnson & Johnson, emphasized that TAR-200 represents a significant advancement in drug delivery for bladder cancer treatment. This recognition by the FDA highlights the company's commitment to transforming treatment options for bladder cancer, particularly for cases where conventional therapies have failed.

The New Drug Application is supported by promising results from the Phase 2b SunRISe-1 clinical trial. The study revealed an 82.4% complete response rate, with more than half of the patients remaining cancer-free for at least a year post-treatment. The adverse reactions observed were mostly mild to moderate, with no systemic adverse effects reported. Common reactions included increased frequency of urination, discomfort during urination, urinary tract infections, urgency to urinate, blood in urine, non-infective cystitis, and urinary tract pain. These findings were shared at the American Urological Association's Annual Meeting in April 2025.

Historically, treatment options for HR-NMIBC patients have remained largely unchanged for over four decades, especially for those who do not respond to initial BCG therapy. TAR-200 offers a new approach as the first intravesical drug-releasing system designed to provide continuous local delivery of cancer medication to the bladder. The device remains in the bladder for three weeks per treatment cycle and can be inserted by a healthcare professional in an outpatient setting in less than five minutes, eliminating the need for general anesthesia or prolonged monitoring.

TAR-200, currently under investigation, is part of Johnson & Johnson's efforts to address the unmet needs in bladder cancer treatment. It is being evaluated in various clinical trials, including Phase 2 and Phase 3 studies, targeting both non-muscle invasive and muscle-invasive bladder cancers.

The SunRISe-1 study, particularly Cohort 2, is a Phase 2b trial focusing on the safety and efficacy of TAR-200 as a monotherapy for patients with BCG-unresponsive HR-NMIBC with carcinoma in situ, who are either unsuitable for or choose not to undergo radical cystectomy. The study's primary goal is to achieve a complete response at any point, with secondary objectives including the duration of response, safety, overall survival, and quality of life.

High-risk non-muscle invasive bladder cancer is more likely to recur and progress to muscle-invasive cancer compared to low-risk forms. HR-NMIBC is characterized by the presence of carcinoma in situ and large or multiple tumors. BCG, a weakened form of tuberculosis bacteria, is traditionally used in treatment, but some patients become unresponsive to it. For these patients, radical cystectomy is often recommended, a procedure that significantly impacts quality of life and carries a notable risk of morbidity and mortality. Given the age of many HR-NMIBC patients, radical cystectomy may not be a suitable option for all.

Johnson & Johnson continues to leverage its expertise in healthcare innovation, aiming to develop treatments that are both effective and less invasive, ultimately enhancing patient outcomes.