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

Phase 2b SunRISe-1 trial findings supported the FDA to grant priority review to TAR-200 in BCG-unresponsive high-risk NMIBC with carcinoma in situ.

The FDA has granted priority review to the new drug application for TAR-200 for patients with Bacillus Calmette-Guérin (BCG)-unresponsive high-risk non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS), with or without papillary tumors, according to a press release from Johnson & Johnson.¹

The submission was supported by results from the phase 2b SunRISe-1 trial (NCT04640623), which were previously reported at the 2025 American Urological Association Annual Meeting.^2,3

Of the 85 patients enrolled in cohort 2 of the trial, the complete response (CR) rate was 82.4% (95% CI, 72.6%-89.8%). Additionally, 52.9% of patients maintained a CR at 1 year.

The median duration of response was 25.8 months (95% CI, -not estimable), and at 12 months, 86.6% (95% CI, 76.6%-92.6%) of patients were cystectomy-free. Overall, the treatment was well-tolerated and the most common adverse effects being mild urinary symptoms.

A total of 83.5% of patients had treatment-related adverse effects (TRAEs), most of which were low-grade, with 12.9% reporting grade 3 or higher TRAEs, and 5.9% had serious TRAEs. Treatment discontinuation due to TRAEs occurred in 3.5% of patients, and none were due to treatment-related deaths.

"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, Johnson & Johnson Innovative Medicine, said in the press release. "The FDA priority review for TAR-200 underscores our mission to fundamentally change the way urologists treat certain types of bladder cancer."

Additional SunRISE-1 Efficacy Data

The SunRISE-1 trial assessed TAR-200 in patients with BCG-unresponsive, high-risk NMIBC. Those with carcinoma in situ with or without papillary disease were randomly assigned to cohorts 1 to 3. If they had papillary disease only, they were assigned to cohort 4.

All patients in cohort 4 were given TAR-200 monotherapy and needed to have persistent or recurrent disease within 12 months of completing BCG, and have been unresponsive to BCG without receiving radical cystectomy.

In cohort 4, the median patient age was 71.0 years, 71.2% were male, 86.5% were White, and 94.2% had an ECOG performance status of 0. Of note, 55.8% of patients were former smokers, 13.5% were current smokers, and 30.8% were never smokers.

Stage Ta or T1 disease was noted in 59.6% vs 40.4%, the median number of prior doses was 12, and the median time from the last dose of BCG to diagnosis of high-grade papillary disease was 2.8 months.

For patients with Ta disease, the 6-month disease-free survival (DFS) was 85.7% (95% CI, 66.3%-94.4%), and the 9-month DFS was 82.1% (95% CI, 62.3%-92.1%). For those with T1 disease, the 6- and 9-month DFS rates were 84.7% (95% CI, 59.7%-94.8%) and 79.4% (95% CI, 54.0%-91.7%), respectively.

The median progression-free survival (PFS) and the median overall survival (OS) were not estimable. At 6 months, the OS rate was 95.6% (95% CI, 83.5%-98.9%), and at 9 months it was 98.0% (95% CI, 86.4%-99.7%). Overall, 1 patient experienced disease progression to MIBC.

The most common any-grade TRAEs included dysuria (40.4%), pollakiuria (30.8%), micturition urgency (26.9%), urinary tract infection (23.1%), and hematuria (13.5%).

Serious TRAEs were noted in 5.8% of patients, and TRAEs leading to discontinuation of treatment occurred in 7.7% of patients, there were no treatment-related deaths reported.

“DFS rates were consistently high across both [patients with] high-grade Ta and T1 [disease], indicative of this [intravesical drug–releasing] system, [TAR-200], increasing the penetration of the drug [gemcitabine] across the bladder wall,” Felix Guerrero-Ramos, MD, PhD, an attending urologist at Hospital Universitario 12 de Octubre in Madrid, Spain, the lead study author, said in a presentation of the data at ESMO.

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://tinyurl.com/4v6c4swz
2. Johnson & Johnson’s TAR-200 monotherapy demonstrates highest complete response rate with sustained clinical benefits in patients with certain types of bladder cancer. News release. April 26, 2025. Accessed July 17, 2025. https://tinyurl.com/26dpjkek
3. Guerrero-Ramos F, Jacob JM, Van der Heijden MS, et al. TAR-200 monotherapy in patients with bacillus Calmette-Guerin–unresponsive papillary disease–only high-risk non–muscle-invasive bladder cancer: first results from Cohort 4 of SunRISe-1. Presented at: 2025 AUA Annual Meeting; April 26-29, 2025; Las Vegas, NV.