FDA Grants Priority Review to TAR-200 for Bladder Cancer Subset

FDA grants priority review to TAR-200 for high-risk bladder cancer unresponsive to BCG: © stock.adobe.com.

The U.S. Food and Drug Administration (FDA) has granted priority review to the new drug application for TAR-200, a gemcitabine-releasing intravesical system, for patients with high-risk non-muscle invasive bladder cancer that is unresponsive to Bacillus Calmette-Guérin (BCG) and includes carcinoma in situ, with or without papillary tumors.

This was announced in a news release from Johnson & Johnson.

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

Data from the phase 2b SunRISe-1 trial supported the FDA submission for TAR-200; data showed a complete response rate of 82.4% in patients with high-risk non-muscle invasive bladder cancer unresponsive to BCG. Among those who responded, 52.9% stayed cancer-free for at least one year. As of March 2025, the median duration of response was 25.8 months, with many patients remaining cancer-free for over two years without needing retreatment. At 12 months, 86.6% of responders had not undergone bladder removal surgery.

As per the news release, findings show that TAR-200 offers a highly effective and durable treatment option for patients with certain types of BCG-unresponsive high-risk non-muscle invasive bladder cancer.

Side effects were mostly mild or moderate and included frequent urination, painful urination, urinary tract infections, urgency, blood in the urine, bladder inflammation, and urinary pain. No systemic side effects were reported. Results were shared during a plenary session at the April 2025 American Urological Association Annual Meeting.

Cohort 2 of the SunRISe-1 trial was the primary source of clinical data supporting the FDA submission for TAR-200. This single-arm, open-label phase 2b study evaluated TAR-200 as monotherapy in patients with BCG-unresponsive high-risk non–muscle invasive bladder cancer, specifically those with carcinoma in situ with or without papillary tumors, who were either ineligible for or opted against radical cystectomy. The study’s primary end point was complete response at any time point, while secondary endpoints included duration of response, safety, overall survival, and quality of life.

Despite limited progress in more than 40 years for high-risk non–muscle-invasive bladder cancer, TAR-200 offers a new option as the first intravesical drug releasing system designed to deliver sustained, localized treatment directly into the bladder, as per the news release. The device stays in place for three weeks per cycle and is inserted by a healthcare professional using a co-packaged catheter in an outpatient setting, typically taking less than five minutes. No general anesthesia, post-insertion monitoring, or immediate restrictions are required after placement.

In December 2023, the FDA granted breakthrough therapy designation for TAR-200 in adults with BCG-unresponsive disease and carcinoma in situ who cannot or choose not to have their bladder surgically removed. The following month, a new drug application was submitted through the FDA’s Real-Time Oncology Review pathway. Ongoing clinical trials — SunRISe-1, -3 and -5 in non–muscle-invasive bladder cancer and SunRISe-4 in muscle-invasive bladder cancer — are evaluating its safety and efficacy.

High-risk non-muscle invasive bladder cancer is a form of bladder cancer that has a higher chance of returning or spreading beyond the bladder’s inner lining compared to lower-risk types. This form accounts for 15- to 44% of all non-muscle-invasive bladder cancers and often involves aggressive features like large tumors, multiple growths, and carcinoma in situ. For patients who don’t respond to BCG treatment, removing the bladder is usually advised and can lead to more than 90% survival specific to the cancer if done before it invades the muscle. Because this disease mostly affects older adults, many may not be able or willing to undergo this major surgery.

References

1. “Johnson & Johnson receives U.S. FDA Priority Review for TAR-200 NDA in high-risk non-muscle invasive bladder cancer,” Johnson & Johnson news release, July 17, 2025.
2. “Johnson & Johnson’s TAR-200 monotherapy demonstrates highest complete response rate with sustained clinical benefits in patients with certain types of bladder cancer,” Johnson & Johnson news release, April 26, 2025.

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