The FDA approved gemcitabine intravesical system (Inlexzo) for BCG-unresponsive non-muscle invasive bladder cancer with carcinoma in situ, demonstrating an 82% complete response rate in the SunRISe-1 trial. This approval provides a critical treatment option for patients who have exhausted BCG therapy and face limited alternatives beyond radical cystectomy.
Drug Profile & Mechanism
- Novel delivery system: Intravesical device delivering 225 mg gemcitabine with 3-week indwelling period
- Target population: Adults with BCG-unresponsive NMIBC with CIS ± papillary tumors post-TURBT
- Mechanism: Nucleoside analog antimetabolite delivered directly to bladder tissue via sustained-release system
- Manufacturer: Janssen Biotech, Inc.
- Co-packaged components: Urinary catheter and stylet for insertion
Regulatory Milestone Details
- Approval pathway: Priority review with breakthrough therapy designation
- Supporting trial: SunRISe-1 Cohort 2 (NCT04640623) single-arm study, n=83 patients
- Primary endpoints: Complete response rate (82%, 95% CI: 72-90%) and duration of response
- Duration of response: 51% of complete responders maintained response ≥12 months
- Review facilitation: Real-Time Oncology Review pilot program and Assessment Aid utilized
Practice Implications
- Treatment schedule: Initial phase every 3 weeks × 6 months (8 doses), then maintenance every 12 weeks × 18 months (6 doses)
- Monitoring requirements: Cystoscopy and urine cytology every 12 weeks for first 2 years, then cystoscopy at least every 24 weeks
- Mandatory assessments: Biopsies required at 24 and 48 weeks after treatment initiation
- Clinical positioning: Treatment option for BCG-unresponsive patients who may otherwise require radical cystectomy
Development Status & Next Steps
- Current status: Approved September 9, 2025 and commercially available
- Prescribing information: Available on Drugs@FDA website
- Safety monitoring: Key warnings include perforated bladder contraindication, MRI safety precautions, and embryo-fetal toxicity
- Treatment discontinuation: Until persistent/recurrent high-grade NMIBC, disease progression, or unacceptable toxicity
Source: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-gemcitabine-intravesical-system-non-muscle-invasive-bladder-cancer
