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FDA approves gemcitabine intravesical system for BCG-unresponsive non-muscle invasive bladder cancer

On September 9 2025, the U.S Food and Drug Administration (FDA) approved gemcitabine intravesical system (formerly TAR-200) for adults with Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.1 Whilst intravesical strategies have been part of the NMBIC armamentarium for almost half a century, approximately 30-50% of patients will progress on treatment, highlighting a critical unmet need for effective, well-tolerated therapies that can delay or avoid radical cystectomy.2

The newly approved system delivers 225 mg of gemcitabine via a sustained-release intravesical implant, designed to remain indwelling for three weeks before removal, and administered in a brief office procedure, without anesthesia. Treatment consists of up to eight doses at three-week intervals over six months, followed by maintenance every 12 weeks for up to 18 months, unless progression, persistent disease, or unacceptable toxicity occurs.1

The approval is based on findings from cohort 2 of the Phase IIb SunRISe-1 trial (NCT04640623), which assessed the gemcitabine intravesical system as a monotherapy in patients with BCG-unresponsive with CIS. 83 patients were enrolled in the cohort and a confirmed complete response (CR) rate of 82.4% (95% CI, 72.6-89.8) was reported. 12.9% of patients in the cohort additionally reported grade ≥3 treatment-related adverse events (AEs), which 5.9% reporting serious treatment-related AEs.3

By offering a non-surgical option in patients with BCG-unresponsive NMIBC, this therapy addresses a substantial unmet need for durable disease control, particularly in those who are poor candidates for radical cystectomy or have previously exhausted standard intravesical regimens.

References:

  1. Center for Drug Evaluation and Research. FDA approves gemcitabine intravesical system for non-muscle invasive bladder cancer [Internet]. FDA; [cited 2025 Sep 9]. Available from: https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancerhematologic-malignancies-approval-notifications
  2. Saum Ghodoussipour, Bivalacqua T, Bryan RT, Li R, Mir MC, Palou J, et al. A Systematic Review of Novel Intravesical Approaches for the Treatment of Patients with Non–muscle-invasive Bladder Cancer. European Urology. 2025 Apr 1
  3. Daneshmand S, Van, Jacob JM, Guerrero-Ramos F, Bögemann M, Simone G, et al. TAR-200 for Bacillus Calmette-Guérin–Unresponsive High-Risk Non–Muscle-Invasive Bladder Cancer: Results From the Phase IIb SunRISe-1 Study. Journal of Clinical Oncology. 2025 Jul 30