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Thomas Flaig, MD

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First Gene Therapy for High-Risk, Non–Muscle-Invasive Bladder Cancer Approved by the FDA

By: JNCCN 360 Staff
Posted: Monday, December 19, 2022

On December 16, the U.S. Food and Drug Administration (FDA) approved nadofaragene firadenovec-vncg (Adstiladrin), a nonreplicating adenoviral vector–based gene therapy, in the treatment of adults with high-risk bacillus Calmette-Guérin (BCG)-unresponsive non–muscle-invasive bladder cancer with carcinoma in situ with or without papillary tumors. This type of cancer is associated with high rates of recurrence (between 30%–80%) and risk of progression to invasive and metastatic cancer. This application was granted Priority Review, Breakthrough Therapy, and Fast Track designations.

The safety and effectiveness of nadofaragene firadenovec were evaluated in a multicenter clinical study that included 157 patients with high-risk BCG-unresponsive non–muscle-invasive bladder cancer, 98 of whom had BCG-unresponsive carcinoma in situ with or without papillary tumors and could be evaluated for response. Patients received the gene therapy once every 3 months for up to 12 months or until unacceptable toxicity to therapy or recurrent high-grade non–muscle-invasive bladder cancer. Overall, 51% of enrolled patients treated with nadofaragene firadenovec achieved a complete response. The median duration of response was 9.7 months. A total of 46% of responding patients remained in complete response for at least 1 year.

Nadofaragene firadenovec is administered once every 3 months into the bladder via a urinary catheter. The most common adverse reactions associated with this gene therapy included bladder discharge, fatigue, bladder spasm, urinary urgency, hematuria, chills, fever, and painful urination.


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