Open Access

ARTICLE

Safety of nadofaragene firadenovec-vncg: review of data from phase 2 and phase 3 studies

Badrinath R. Konety^1,*, Yair Lotan², Amanda Myers³

1 Department of Urology, University of Minnesota and Allina Health Cancer Institute, Minneapolis, MN 55407, USA
2 Department of Urology, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA
3 Department of Urology, University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA

* Corresponding Author: Badrinath R. Konety. Email:

Canadian Journal of Urology 2025, 32(1), 29-36. https://doi.org/10.32604/cju.2025.064710

Received 09 December 2024; Accepted 21 February 2025; Issue published 20 March 2025

Abstract

Introduction: Non–muscle-invasive bladder cancer (NMIBC) is a common malignancy worldwide. While Bacillus Calmette-Guérin (BCG) is standard of care for treatment for most patients with high-risk NMIBC, many will either not respond to BCG initially or will eventually develop BCG-unresponsive disease. A treatment option in BCG-unresponsive disease is nadofaragene firadenovec-vncg (Adstiladrin), a nonreplicating adenoviral vector–based gene therapy approved by the US Food and Drug Administration (FDA) for the treatment of adults with high-risk BCG-unresponsive NMIBC with carcinoma in situ with or without papillary tumors. Objective: To review safety outcomes of participants who received the FDA-approved dose of nadofaragene firadenovec (3 × 10¹¹ vp/mL) across phase 2 (NCT01687244) and phase 3 (NCT02773849) studies. Methods: Data from the phase 2 and phase 3 studies were collected and analyzed. The findings were reported using descriptive statistics to summarize the key outcomes observed across studies. Results: Common adverse events (AEs) among nadofaragene firadenovec recipients were leakage of fluid around the urinary catheter, fatigue, bladder spasm, chills, dysuria, and micturition urgency. Most study drug–related AEs were mild and localized, with no grade 4 or 5 study drug–related AEs observed in either study. Study drug–related AEs were generally transient, with most study drug–related AEs having a median duration of ≤2.0 days in the phase 3 study. Discontinuation rates due to study drug–related AEs were low, with none (0%) in the phase 2 study and three (1.9%) in the phase 3 study. No specific postmarketing surveillance was required by the FDA besides routine pharmacovigilance monitoring; no new real-world safety signals have been observed. Conclusion: Nadofaragene firadenovec demonstrated a favorable and tolerable safety profile across its clinical study program, allowing for broad patient selection among those with high-risk BCG-unresponsive NMIBC.

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