Real-world outcomes of nadofaragene firadenovec for BCG-unresponsive non-muscle-invasive bladder cancer: A multicenter experience.

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.