Gemcitabine Intravesical System Receives FDA OK for NMIBC

The FDA's approval includes patients with and without paillary tumors.

The FDA has granted approval to gemcitabine intravesical system (Inlexzo) for the treatment of adults with Bacullus Calmette-Guéruin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC).^1,2

This approval includes patients with carcinoma in situ (CIS) with or without papillary tumors. Gemcitabine intravesical system comes packaged with a urinary catheter and stylet used for insertion via urinary catheter into the bladder.

“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 [gemcitabine intravesical system],” said Sia Daneshmand, MD, a principal investigator for SunRISe-1 as well as professor of urology and director of urologic oncology at the Norris Comprehensive Cancer Center, Keck School of Medicine of University of Southern California. “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.”

Gemcitabine Intravesical System’s Efficacy: Then and Now

The efficacy of gemcitabine intravesical system was assessed in cohort 2 of the single-arm, multicenter phase 2b SunRISe-1 trial (NCT04640623), at which point the drug was referred to as TAR-200. The trial enrolled 83 patients with BCG-unresponsive NMIBC after transurethral resection.

Gemcitabine intravesical system was granted priority review in July 2025 based on results from the trial shared in a plenary session at the 2025 American Urological Association (AUA) Annual Meeting in April and published in The Journal of Urology.^3,4

Per data published in the FDA’s and Johnson & Johnson’s announcements on the drug’s approval, updated results demonstrate an 82% complete response (CR; 95% CI, 72%-90%), with 51% of those with a CR maintaining the CR for at least 12 months.

As of the March 31, 2025 data cutoff, investigator-assessed and centrally confirmed CR for cohorts 1-3 of SunRISe-1 were observed in 83.5% (95% CI, 73.9-90.7) and 82.4% (95% CI, 72.6-89.8) of all patients (n = 85), respectively. At 12 months, the observed CR rate was 45.9%, with an estimated median duration of response (DOR) of 25.8 months (95% CI, 8.3-NE). Of note, 56.2% (95% CI, 43.4-67.1) of patients experienced a DOR of at least 1 year.

SunRISe-1’s primary end point for cohort 2 was CR, with a secondary end point of DOR.

Additionally, 11 patients received therapy for at least 2 years, and 9 remain in response to TAR-200. At baseline, patients had high mean Global Health Status (GHS) and Physical Functioning (PF) scores, and scores remained stable during treatment and did not change by a clinically meaningful interval.

The median age of enrolled patients was 71 years (range, 40-88), 80% of whom were male, and 33% of whom had concurrent papillary disease.

What is Gemcitabine Intravesical System’s Safety Profile?

Of note, the gemcitabine intravesical system’s label includes warnings for patients with a perforated bladder, risk of metastatic bladder cancer with delayed cystectomy, and magnetic resonance imaging (MRI) safety, and embryo-fetal toxicity.¹

With regard to data published in The Journal of Urology, the majority of patients (83.5%) in cohort 2 of SunRISe-1 experienced treatment-related adverse effects (TRAEs) on trial. Most TRAEs were low-grade lower urinary tract AEs. Eleven patients (12.9%) had grade 3 or higher TRAEs, 5 (5.9%) had serious TRAEs, and 3 (3.5%) discontinued treatment due to toxicity. No deaths related to treatment with TAR-200 occurred.⁴

How is Gemcitabine Intravesical System Administered?

Gemcitabine intravesical system is administered at 225 mg via catheter, with a 3-week indwelling period after each insertion before the catheter is removed.¹ The system is inserted every 3 weeks for as many as 6 months, amounting to 8 doses, then once every 12 weeks for as many as 18 months, amounting to 6 doses, or until persistent or recurrent high-grade NMIBC, disease progression, or unacceptable toxicity.

References

1. FDA approves gemcitabine intravesical system for non-muscle invasive bladder cancer. FDA. September 9, 2025. Accessed September 9, 2025. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-gemcitabine-intravesical-system-non-muscle-invasive-bladder-cancer
2. U.S. FDA approval of INLEXZO™ (gemcitabine intravesical system) set to transform how certain bladder cancers are treated. Johnson & Johnson. News release. September 9, 2025. Accessed September 9, 2025. https://www.multivu.com/johnson-and-johnson/9342851-en-johnson-and-johnson-fda-approval-inlexzo-gemcitabine-intravesical-system
3. Johnson & Johnson receives U.S. FDA Priority Review for TAR-200 NDA in high-risk non-muscle invasive bladder cancer. FDA. July 17, 2025. Accessed July 17, 2025. https://www.prnewswire.com/news-releases/johnson--johnson-receives-us-fda-priority-review-for-tar-200-nda-in-high-risk-non-muscle-invasive-bladder-cancer-302507112.html
4. Jacob JM, Guerrero-Ramos F, Necchi A, et al. TAR-200 monotherapy in patients with Bacillus Calmette-Guérin–unresponsive high-risk non–muscle-invasive bladder cancer carcinoma in situ: 1-year durability and patient-reported outcomes from SunRISe-1. J Urol. 2025;213(5S2):e2. doi:10.1097/01.JU.0001111604.90306.91.03