Gemcitabine Intravesical System Approved for BCG-Unresponsive Non–Muscle Invasive Bladder Cancer
Expert Perspectives · Lyn Brook · 2025-09-10
The FDA has approved gemcitabine intravesical system (Inlexzo™, Janssen Biotech, Inc.) for the treatment of adults with bacillus Calmette-Guérin (BCG)–unresponsive non–muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS), with or without papillary tumors. This therapy is administered through a co-packaged urinary catheter and stylet designed to deliver gemcitabine directly into the bladder.
Why This Approval Matters
The approval of gemcitabine intravesical system provides a new therapeutic option for patients with BCG-unresponsive NMIBC, a population with limited treatment alternatives and a high risk of disease progression. By delivering gemcitabine locally and maintaining drug exposure in the bladder, this treatment offers a minimally invasive alternative for patients who are often faced with the prospect of radical cystectomy.
Breaking Down the SunRISe-1 Trial
Efficacy was evaluated in Cohort 2 of SunRISe-1 (NCT04640623), a multicenter, single-arm trial that enrolled 83 patients with BCG-unresponsive NMIBC with CIS, with or without papillary tumors, following transurethral resection. Patients were treated with the gemcitabine intravesical system every three weeks for six months, then once every 12 weeks for up to an additional 18 months.
Cystoscopy and urine cytology were used to evaluate tumor status every 12 weeks for the first two years of treatment, with subsequent evaluation by cystoscopy occurring at least every 24 weeks. Mandatory biopsies were carried out at Weeks 24 and 48.
The primary efficacy end points were complete response (CR) at any time, defined as negative findings on cystoscopy (with transurethral resection of bladder tumor /biopsy as needed) and urine cytology, and duration of response (DOR).
Gemcitabine intravesical system produced a CR rate of 82%, and 51% of patients who achieved CR maintained their response for 12 months or longer.
Managing Adverse Events
The prescribing information contains warnings and precautions for patients with a perforated bladder, as administration of the intravesical system in this population could result in systemic exposure. Additional warnings include the risk of progression to metastatic bladder cancer with delayed cystectomy, magnetic resonance imaging (MRI) safety considerations, and embryo-fetal toxicity. Clinicians should monitor for these risks during treatment.
Conclusion
“TAR-200 monotherapy was well tolerated, with a high CR rate, durable responses, and prolonged disease-free survival in patients with BCG-unresponsive high-risk NMIBC,” concluded Siamak Daneshmand, MD, Professor of Urology and Oncology at the University of Southern California (USC) Norris Comprehensive Cancer Center, and colleagues, in their published results of the SunRISe-1 trial, on which approval was based. “TAR-200 monotherapy offered a more favorable risk-benefit profile versus TAR-200 plus cetrelimab or cetrelimab alone in BCG-unresponsive CIS.”
Dosing and Administration
Each gemcitabine intravesical system delivers 225 mg of gemcitabine into the bladder, where it remains indwelling for three weeks prior to removal. The system is inserted once every three weeks for up to six months (eight doses), followed by once every 12 weeks for up to 18 months (six doses), or until evidence of persistent or recurrent high-grade NMIBC, disease progression, or unacceptable toxicity.