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FDA Approves Nadofaragene Firadenovec for High-risk Non-Muscle Invasive Bladder Cancer

The FDA has approved nadofaragene firadenovec-vncg (Adstiladrin) as the first gene therapy for adult patients with high-risk Bacillus Calmette-Guérin (BCG)–unresponsive non–muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS), offering a new treatment option beyond bladder removal surgery.

The FDA's approval of nadofaragene firadenovec-vncg marks a significant milestone in the treatment of high-risk non-muscle invasive bladder cancer (NMIBC) that does not respond to BCG therapy. This gene therapy, evaluated in the phase 3 Study CS-003, showed a complete response rate of 51% in patients with CIS, with or without concomitant high-grade Ta or T1 disease, by 3 months. The median duration of response was 9.7 months, with 46% of patients remaining free of high-grade recurrence at 12 months.
Peter Marks, MD, PhD, highlighted the importance of this approval, stating it addresses a critical need and underscores the FDA's commitment to advancing safe and effective cancer treatments. The study involved 103 adults with BCG-unresponsive, high-risk NMIBC, who received intravesical instillation of nadofaragene firadenovec-vncg every 3 months for up to 12 months or until intolerable toxicity or recurrent high-grade disease.
Safety evaluations in 157 patients revealed that 11% experienced serious toxicities, with common adverse reactions including instillation site discharge, fatigue, and bladder spasm. Despite these, the therapy represents a novel option for patients who have historically faced limited treatment choices beyond radical surgery.
Steven A. Boorjian, MD, emphasized the significance of this approval, noting it as a major advancement in the treatment landscape for BCG-unresponsive NMIBC, providing hope and a new therapeutic avenue for patients.
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Reference News

[1]
FDA Approves Nadofaragene Firadenovec for High-risk ...
onclive.com · Dec 16, 2022

FDA approved nadofaragene firadenovec-vncg, the first gene therapy for high-risk BCG-unresponsive NMIBC with CIS. In a p...

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