FDA approves gemcitabine intravesical system for non-muscle invasive bladder cancer
Regulatory approval: On Sept 9, 2025, the FDA approved gemcitabine intravesical system (Inlexzo; Janssen Biotech) for adults with BCG-unresponsive non-muscle invasive bladder cancer with carcinoma in situ (with or without papillary tumors). Approval was supported by Cohort 2 of the single-arm SunRISe-1 trial (NCT04640623; n=83), which reported an 82% complete response rate (95% CI, 72–90); 51% of responders had duration of response ≥12 months.
FDA approval: gemcitabine intravesical system for BCG-unresponsive NMIBC with CIS
Type: Regulatory approval
On September 9, 2025, the FDA approved gemcitabine intravesical system (Inlexzo, Janssen Biotech, Inc.) for adults with Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS), with or without papillary tumors.
The product is co-packaged with a urinary catheter and stylet for bladder insertion. It delivers 225 mg gemcitabine intravesically, with a 3-week indwelling period after each insertion. The regimen is once every 3 weeks for up to 6 months (8 doses), then once every 12 weeks for up to 18 months (6 doses), or until persistent/recurrent high-grade NMIBC, progression, or unacceptable toxicity.
Supporting evidence
Efficacy was evaluated in Cohort 2 of SunRISe-1 (NCT04640623), a single-arm, multicenter trial enrolling 83 patients with BCG-unresponsive NMIBC with CIS (± papillary tumors) following transurethral resection. Tumor status was assessed by cystoscopy and urine cytology every 12 weeks for 2 years; mandatory biopsies occurred at 24 and 48 weeks. Complete response (CR) was based on central pathology review.
CR rate: 82% (95% CI, 72–90). Among patients achieving CR, 51% had duration of response ≥12 months.
Label warnings/precautions (selected)
Risks include use in perforated bladder, potential metastatic bladder cancer with delayed cystectomy, MRI safety, and embryo-fetal toxicity. Full prescribing information is expected on Drugs@FDA.
The FDA review used RTOR and priority review; the product had breakthrough therapy designation.