FDA Grants Priority Review to TAR-200 for BCG-Unresponsive High-Risk NMIBC

FDA

The FDA has granted priority review to a new drug application (NDA) seeking the approval of TAR-200, an investigational intravesical gemcitabine delivery system, for the treatment of patients with BCG-unresponsive, high-risk non–muscle-invasive bladder cancer (NMIBC) with carcinoma in situ (CIS), with or without papillary tumors.1

The NDA is supported by data from the phase 2b SunRISe-1 trial (NCT04640623), which were presented during a plenary session at the 2025 American Urological Association Annual Meeting. In the trial, TAR-200 demonstrated a complete response (CR) rate of 82.4% among evaluable patients. Notably, 52.9% (95% CI, 72.6%-89.8%) of patients who achieved a CR remained disease free for at least 1 year.

The safety profile of TAR-200 was favorable, with most adverse effects (AEs) being mild or moderate in severity. The most frequently observed AEs reported in at least 10% of patients included pollakiuria, dysuria, urinary tract infection, micturition urgency, hematuria, noninfective cystitis, and urinary tract pain.

"TAR-200 represents an innovation in drug delivery that has not been seen in decades," Yusri Elsayed, MD, MHSc, PhD, global therapeutic area head, Oncology, at Johnson & Johnson Innovative Medicine, stated in a news release. "The FDA priority review for TAR-200 underscores our mission to fundamentally change the way urologists treat certain types of bladder cancer."

SunRISe-1 Trial Breakdown and Enrollment Criteria

Cohort 2 of SunRISe-1enrolled patients with high-risk NMIBC with CIS or tumor in situ, with or without papillary disease, who were unresponsive to BCG.2 Eligible patients were required to have persistent or recurrent disease within 12 months of completing adequate BCG, defined as a minimum of 5 of 6 induction doses and 2 of 3 maintenance doses, or 2 of 6 doses of a second induction course. Patients were also required to be ineligible for or declined radical cystectomy.

Patients received TAR-200 as monotherapy administered transuretherally via a urinary placement catheter. In this cohort, TAR-200 was inserted on Day 0 and dosed every 3 weeks for up to 24 weeks, then every 12 weeks through week 99.

The primary end point was CR rate at any time point. Secondary end points included duration of response (DOR), overall survival (OS), safety, and health-related quality of life.

TAR-200 Within the Bladder Cancer Treatment Paradigm

On January 5, 2024, the FDA granted breakthrough therapy designation to TAR-200 for use in the treatment of patients with BCG-unresponsive, high-risk NMIBC who are not candidates for or opted not to receive radical cystectomy.1

The safety and efficacy of TAR-200 are currently being evaluated across multiple clinical trials. Beyond SunRISe-1, the phase 3 SunRISe-3 study (NCT05714202) is designed to further assess TAR-200 in combination with cetrelimab or as monotherapy in the BCG-unresponsive setting. The phase 3 SunRISe-5 study (NCT05934765) is evaluating TAR-200 in patients with intermediate-risk NMIBC.

Additionally, the phase 2 SunRISe-4 study (NCT04919512) is investigating TAR-200 in combination with cetrelimab in patients with muscle-invasive bladder cancer.

References

1. Johnson & Johnson receives U.S. FDA Priority Review for TAR-200 NDA in high-risk non-muscle invasive bladder cancer. News Release. Johnson & Johnson. July 17, 2025. Accessed July 17, 2025. https://www.jnj.com/media-center/press-releases/johnson-johnson-receives-u-s-fda-priority-review-for-tar-200-nda-in-high-risk-non-muscle-invasive-bladder-cancer
2. A study of TAR-200 in combination with cetrelimab, TAR-200 alone, or cetrelimab alone in participants with non–muscle-invasive bladder cancer (NMIBC) unresponsive to intravesical Bacillus Calmette-Guérin who are ineligible for or elected not to undergo radical cystectomy (SunRISe-1). ClinicalTrials.gov. Updated June 22, 2025. Accessed July 17, 2025. https://clinicaltrials.gov/study/NCT04640623