FDA Grants Priority Review to NDA of TAR-200 in BCG-Unresponsive NMIBC

• The FDA has granted priority review to the new drug application (NDA) for TAR-200 in Bacillus Calmette-Guérin (BCG)-unresponsive high-risk non–muscle-invasive bladder cancer (HR-NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.
• TAR-200 is an intravesical gemcitabine-releasing system.
• Findings from the phase 2b SunRISe-1 study (NCT04640623) support this regulatory decision.

The FDA has granted priority review to the NDA filed for TAR-200 in patients with BCG-unresponsive HR-NMIBC with CIS, with or without papillary tumors.¹

TAR-200, an intravesical gemcitabine-releasing system, achieved an overall complete response (CR) rate of 82.4% (95% CI, 72.6%–89.8%) when given to patients BCG-unresponsive, HR-NMIBC with CIS with or without papillary disease in the phase 2b SunRISe-1 study.² The 12-month CR rate was 45.9%. The Kaplan-Meier–estimated 12- and 24-month CR rates were 52.4% (95% CI, 40.7%–62.8%) and 44.7% (95% CI, 33.1%–55.7%), respectively. The median time to onset of CR was 2.8 months (range, 2.1–8.3) and 95.7% (n = 67/70) of patients achieved a CR within the first 3 months. Additionally, the CR rate was consistent across patient subgroups.

Findings from the phase 2b SunRISe-1 study were presented during the 2025 American Urological Association Annual Meeting and support this FDA’s decision to grant priority review to this NDA of TAR-200.¹

“TAR-200 represents an innovation in drug delivery that has not been seen in decades,” said Yusri Elsayed, MD, MHSc, PhD, Global Therapeutic Area Head, Oncology, Johnson & Johnson Innovative Medicine, in a press release. “The FDA priority review for TAR-200 underscores our mission to fundamentally change the way urologists treat certain types of bladder cancer.”

TAR-200 employs an intravesical drug releasing application which provides sustained delivery of gemcitabine in the bladder. In December 2023, TAR-200 received breakthrough therapy designation from the FDA for the treatment of adult patients with BCG-unresponsive, high-risk NMIBC who are ineligible for or have declined radical cystectomy.³ In January 2025, the NDA was submitted to the FDA seeking the approval of the agent for the treatment of patients with BCG-unresponsive, high-risk NMIBC with CIS, regardless of the presence of papillary tumors.⁴

TAR-200 was assessed in the SunRISe-1 trial which enrolled adult patients with histologically confirmed high-risk NMIBC with CIS with or without papillary disease into cohorts 1,2, and 3.² Patients had an ECOG performance status of 2 or less and were required to have persistent or recurrent disease within 12 months of completion of BCG therapy and be unresponsive to BCG therapy with no plans to undergo a radical cystectomy. Cohort 4 of the trial enrolled patients with papillary-only high-risk NMIBC without CIS.

Patients in cohort 1 were given TAR-200 in combination with cetrelimab and those in cohorts 2 and 3 received TAR-200 or cetrelimab monotherapy, respectively. Patients in cohort 4 also received TAR-200 monotherapy. TAR-200 was administered once every 3 weeks for the first 24 weeks then every 12 weeks through week 96 of treatment.

The primary end point was overall CR rate in cohorts 1, 2, and 3, and key secondary end points included duration of response (DOR), overall survival, safety, tolerability, and health-related quality of life (HRQOL). In cohort 4, the primary end point was disease-free survival.

Additional findings from the SunRISe-1 trial presented at ASCO showed that the median DOR was 25.8 months (95% CI, 8.3–not evaluable). High-risk NMIBC recurrence was seen in 32.9% of responders. Additionally, 5.7% experienced at least T2 disease progression.

At 12 months, the cystectomy-free rate was 86.6% (95% CI, 76.6%–92.6%). The estimated 12- and 24-month DOR probabilities were 56.2% (95% CI, 43.4%–67.1%) and 51.8% (95% CI, 38.7%–63.4%), respectively. The 12-month DOR rate was 52.9%, and 47.1% of responses were ongoing at the data cutoff.

Looking at safety, most treatment-emergent adverse events were grade 1 or 2 in cohort 2, and they resolved after a median of 3.1 weeks. A total of 5 patients had at least 1 serious treatment-related adverse effect (TRAE), and 3.5% of patients discontinued TAR-200 monotherapy due to TRAEs. No treatment-related deaths were reported.

Further, any-grade and grade 3 or higher TRAEs occurred at rates of 83.5% and 12.9%, respectively. Any-grade TRAEs included pollakiuria (43.5%), dysuria (40.0%), and micturition urgency (24.7%), and grade 3 or higher TRAEs consisted of urinary tract pain (4.7%), bladder pain (2.4%), and urinary tract infection (1.2%).

REFERENCES:

1. Johnson & Johnson receives U.S. FDA priority review for TAR-200 NDA in high-risk non-muscle invasive bladder cancer. News release. FDA. July 17, 2025. Accessed July 17, 2025. https://tinyurl.com/33rt89va

2. Jacob JM, Guerrero-Ramos F, Necchi A, et al. TAR-200 monotherapy in patients with Bacillus Calmette-Guerin–unresponsive high-risk non–muscle-invasive bladder cancer carcinoma in situ: 1-year durability and patient-reported outcomes from SunRISe-1. Presented at: American Urological Association Annual Congress; April 26-29, 2025; Las Vegas, NV. P2s.

3. Johnson & Johnson’s investigational TAR-200 granted US FDA breakthrough therapy designation for the treatment of high-risk non-muscle-invasive bladder cancer. News release. Johnson & Johnson. December 4, 2023. Accessed July 17, 2025. https://tinyurl.com/3bbnwjna

4. New drug application initiated with US FDA for TAR-200, the first and only intravesical drug releasing system for patients with BCG-unresponsive high-risk non-muscle-invasive bladder cancer. News release. Johnson & Johnson. January 15, 2025. Accessed July 17, 2025. https://tinyurl.com/yc7a9d4e