FDA approves gemcitabine intravesical system for BCG-unresponsive NMIBC

On September 9, 2025, the FDA approved gemcitabine intravesical system (Inlexzo; formerly TAR-200) for adult patients with BCG-unresponsive non–muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.¹

According to Johnson & Johnson, Inlexzo is the first and only intravesical drug releasing system to provide extended local delivery of a cancer medication into the bladder. The system is designed to remain in the bladder for 3 weeks per treatment cycle for up to 14 cycles.

With this approval, Inlexzo becomes an additional treatment option for patients following unsuccessful BCG therapy and for patients refusing or ineligible for radical cystectomy.

“I see many patients that ultimately become BCG-unresponsive and often face life-altering bladder removal. These patients now may be ideal candidates for newly approved Inlexzo,” said SunRISe-1 principal investigator Sia Daneshmand, MD, professor of urology and director of urologic oncology at the Norris Comprehensive Cancer Center, Keck School of Medicine of University of Southern California, Los Angeles, in the news release.¹ “In my experience, Inlexzo is well-tolerated and delivers clinically meaningful results. This will change the way we treat appropriate patients that haven't responded to traditional therapy.”

Data on Inlexzo

The approval is supported by data from cohort 2 of the phase 2b SunRISe-1 trial (NCT04640623), which showed a complete response (CR) rate of 82% (95% CI, 72 to 90) in patients with BCG-unresponsive NMIBC with CIS. Further, 51% of patients remained in CR for at least 1 year.

Overall, the SunRISe-1 trial is assessing the safety and efficacy of Inlexzo across 4 patient cohorts. Cohort 2 of the study, which is assessing Inlexzo monotherapy, included 85 patients with BCG-unresponsive NMIBC with CIS with or without papillary tumors. The median age of patients was 71 years, and the majority of patients were male (80.0%) and White (87.1%).

The primary end point in cohort 2 of the study was centrally assessed CR rate at any time point. Secondary end points included duration of response (DOR), overall survival (OS), changes from baseline in patient-reported outcomes, and safety and tolerability.

Data from the trial, which were recently published in the Journal of Clinical Oncology,² showed that the median onset to CR was 2.8 months, with 96% of patients achieving a CR within the first 3 months. The 3-, 6-, and 12-month CR rates were 78.8% (95% CI, 68.6 to 86.9), 58.8% (95% CI, 47.6 to 69.4), and 45.9% (95% CI, 35.0 to 57.0), respectively. According to the authors, high CR rates were observed across all clinically relevant subgroups, including in those with and without concurrent papillary disease (82.1% to 82.5%, respectively).

Data also showed a median DOR of 25.8 months (95% CI, 8.3 to NE).

Regarding safety, the most common (15% or greater) adverse events (AEs) included urinary frequency, urinary tract infection, dysuria, micturition urgency, decreased hemoglobin, increased lipase, urinary tract pain, decreased lymphocytes, hematuria, increased creatinine, increased potassium, increased aspartate aminotransferase, decreased sodium, bladder irritation, and increased alanine transaminase.

Grade 3 or higher AEs were reported in 12.9% of patients, with the most frequent being urinary tract pain (4.7%). Serious TRAEs occurred in 5.9% of patients.

In addition to cohort 2, the SunRISe-1 trial also assessed the safety and efficacy of Inlexzo in combination with cetrelimab (cohort 1; n = 53) and cetrelimab monotherapy (cohort 3; n =28) in patients with BCG-unresponsive high-risk NMIBC with CIS. The trial also includes a fourth cohort assessing Inlexzo monotherapy in patients with BCG-unresponsive papillary disease-only high-risk NMIBC (n = 52).

According to the authors, “TAR-200 monotherapy demonstrated a more favorable risk-benefit profile compared with the combination of TAR-200 plus cetrelimab or cetrelimab monotherapy in this disease setting.”

Additional Information about Inlexzo

Inlexzo is designed for patients seeking bladder preservation. The treatment is placed in the bladder using a co-package urinary catheter and stylet and can be delivered in an outpatient setting without the need for general anesthesia.

Leading up to approval, the FDA granted Breakthrough Therapy designation, real-time oncology review, and priority review to Inlexzo in this disease setting.

“When we acquired this novel therapy in 2019, our ambition was to give patients with bladder cancer a renewed sense of hope and belief,” noted Jennifer Taubert, Executive Vice President, Worldwide Chairman, Innovative Medicine, Johnson & Johnson, in the news release.¹ "In an area that has seen little progress for more than 40 years, Inlexzo delivers a first-of-its-kind breakthrough innovation with a bright future ahead.”

REFERENCES

1. U.S. FDA approval of INLEXZO (gemcitabine intravesical system) set to transform how certain bladder cancers are treated. News release. Johnson & Johnson. September 9, 2025. Accessed September 9, 2025. https://www.jnj.com/media-center/press-releases/u-s-fda-approval-of-inlexzo-gemcitabine-intravesical-system-set-to-transform-how-certain-bladder-cancers-are-treated

2. Daneshmand S, Heijden MSV, Jacob JM, et al. TAR-200 for Bacillus Calmette-Guérin-unresponsive high-risk non-muscle-invasive bladder cancer: Results from the phase IIb SunRISe-1 study. J Clin Oncol. 2025:101200JCO2501651. doi:10.1200/JCO-25-01651