FDA Prioritizes Review of TAR-200 for High-Risk NMIBC Treatment

On July 17, 2025, the U.S. Food and Drug Administration (FDA) granted priority review status to TAR-200, a novel treatment for patients with Bacillus Calmette-Guérin (BCG)-unresponsive high-risk non-muscle invasive bladder cancer (NMIBC) featuring carcinoma in situ (CIS). This decision follows the encouraging results from the Phase 2b SunRISe-1 trial, which was discussed at the 2025 American Urological Association Annual Meeting.

TAR-200, developed by Johnson & Johnson, employs an innovative drug delivery system aimed at enhancing the therapeutic efficacy of gemcitabine, a chemotherapy agent. According to Dr. Yusri Elsayed, Global Therapeutic Area Head for Oncology at Johnson & Johnson, the FDA's prioritization reflects the company's commitment to revolutionizing treatment protocols for bladder cancer. Dr. Elsayed stated, "TAR-200 represents an innovation in drug delivery that has not been seen in decades."

The SunRISe-1 trial involved a diverse cohort of patients who had shown unresponsiveness to BCG therapy. In particular, cohort 4 of the trial included patients with carcinoma in situ who had not undergone radical cystectomy. Among these patients, the median age was 71 years, with a male predominance of 71.2%. Notably, 59.6% of subjects presented with Stage Ta or T1 disease. The trial indicated impressive six-month disease-free survival (DFS) rates of 85.7% for those with Ta disease and 84.7% for those with T1 disease. Furthermore, the overall survival (OS) rates were reported as 95.6% at six months and 98.0% at nine months, indicating robust treatment efficacy.

Adverse effects associated with TAR-200 included dysuria (40.4%), pollakiuria (30.8%), and urinary tract infections (23.1%). Serious treatment-related adverse effects were observed in 5.8% of participants, while 7.7% of patients discontinued treatment due to adverse effects. Importantly, there were no treatment-related fatalities reported, which suggests a favorable safety profile for this new therapy.

Dr. Felix Guerrero-Ramos, an attending urologist at Hospital Universitario 12 de Octubre in Madrid and lead author of the study, noted that the high DFS rates across both Ta and T1 disease highlight TAR-200's potential to improve drug penetration across the bladder wall, enhancing therapeutic outcomes for patients.

The FDA’s priority review is a pivotal step toward making TAR-200 available for patients suffering from this challenging cancer type. The implications of this review extend beyond patient care, as it may influence future drug development and regulatory practices within oncology. As the medical community awaits the FDA's final decision, the results from the SunRISe-1 trial underscore a significant advancement in the management of high-risk NMIBC, potentially setting a new standard in treatment options.

As this situation evolves, stakeholders within the cancer treatment landscape—including oncologists, researchers, and patients—remain hopeful that TAR-200 will provide a much-needed alternative for those with limited options after traditional BCG therapy. The field will continue to monitor the FDA’s review process and subsequent decisions regarding TAR-200's approval.