Abstract
BACKGROUND: Nadofaragene firadenovec-vncg received US Food and Drug Administration approval for bacille Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer (NMIBC) based on prospective efficacy and safety results. However, postmarketing data are lacking. This study evaluates outcomes in a real-world setting. METHODS: We analyzed data for patients treated at Mayo Clinic from November 2023 through December 2024. Outcomes included complete response (CR) rate, duration of response, adverse events, and high-grade recurrence-free (HGRFS), cystectomy-free, and overall survival. RESULTS: Forty-six patients were treated with nadofaragene firadenovec; 3 with pending posttreatment cystoscopy were excluded. Of 24 evaluable patients with carcinoma in situ with/without papillary disease, 79% had a CR at 3 months. The median response duration was not reached; 68% (13/19) of responders still had CRs with median follow-up of 9.3 months. Of 19 patients with papillary-only disease (median follow-up of 7.6 months), HGRFS at 3, 6, and 12 months was 68%, 53%, and 26%, respectively. With median follow-up of 13.9 months, cystectomy-free and overall survival were 93% and 95%, respectively. Progression occurred in 5 patients. The most common adverse events were grade 1-2 bladder spasms (61%) and failure to fully retain the instillation (33%). Four patients had grade 3 events; there were no grade 4 or 5 events. Limitations include the small sample size and limited follow-up. CONCLUSIONS: Real-world data confirm the efficacy and safety of nadofaragene firadenovec, clarifying its role in the management of BCG-unresponsive NMIBC.