FDA approves nadofaragene firadenovec for high-risk NMIBC

Nadofaragene firadenovec is a non-replicating adenoviral vector based gene therapy.

The FDA has approved nadofaragene firadenovec-vncg (Adstiladrin) for the treatment of patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.1

The FDA based its decision on a study that included 157 patients with high-risk BCG-unresponsive NMIBC. Of these patients, 98 had BCG-unresponsive CIS with or without papillary tumors and could be evaluated for response.

Overall, nadofaragene firadenovec, which is a non-replicating adenoviral vector based gene therapy, induced a complete response in 51% of patients. The median response duration was 9.7 months. Of the responders, 46% remained in complete response at 1 year.

“This approval provides healthcare professionals with an innovative treatment option for patients with high-risk non-muscle invasive bladder cancer that is unresponsive to BCG therapy,” Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, stated in a press release. “Today’s action addresses an area of critical need. The FDA remains committed to facilitating the development and approval of safe and effective cancer treatments.”

According to the FDA, the most common adverse reactions associated with Adstiladrin included bladder discharge, fatigue, bladder spasm, urinary urgency, hematuria, chills, fever, and painful urination.


1. FDA Approves First Gene Therapy for the Treatment of High-Risk, Non-Muscle-Invasive Bladder Cancer. Published online December 16, 2022. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treatment-high-risk-non-muscle-invasive-bladder-cancer?utm_medium=email&utm_source=govdelivery

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