The FDA has approved Adstiladrin (nadofaragene firadenovec-vncg) for the treatment of adult patients with high-risk Bacillus Calmette-Guérin–unresponsive non–muscle invasive bladder cancer (NMIBC) with carcinoma in situ with or without papillary tumors.1
About Adstiladrin
Adstiladrin is a non-replicating adenoviral vector–based gene therapy. Adstiladrin carries a copy of the gene for interferon alfa-2b into bladder cancer cells. This may help the body’s immune system recognize the cancer cells and kill them. Adstiladrin is given in a solution that is placed directly into the bladder using a thin tube called a catheter.
The approval was based on clinical study CS-003 which evaluated the safety and effectiveness of Adstiladrin in 103 adults with BCG-unresponsive, high-risk, NMIBC with or without papillary tumors after transurtheral resection.
Before treatment with Adstiladrin, all patients underwent transurethral resection of bladder tumor to eliminate all resectable disease. Aldstiladrin was administered via intravesical instillation once every 3 months for up to 12 months.
Adstiladrin elicited a complete response rate of 51% by 3 months in patients with CIS with or without concomitant high-grade Ta or T1 disease.2 The median duration of response was 9.7 months (range, 3-52+).
The most common side effects reported in more than 10% of patients who received the therapy were instillation site discharge (33%), fatigue (24%), chills (16%), fever (15%), bladder spasm (20%), and bloody or painful urination (17%).
Reference
1. FDA approves first gene therapy for the treatment of high-risk, non-muscle-invasive bladder cancer. News release. FDA. December 16, 2022. Accessed December 16, 2022. https://bit.ly/3hANetF
2. Nadofaragene firadenovec-vncg (Adstiladrin). Prescribing information; Ferring Pharmaceuticals; 2022. Accessed December 16, 2022. https://www.ferringusa.com/wp-content/uploads/sites/12/2022/12/ADSTILADRIN_pi.pdf
3. Ferring receives approval from US FDA for Adstiladrin for high-risk, BCG-unresponsive non-muscle invasive bladder cancer. News release. Ferring Pharmaceuticals. December 16, 2022. Accessed December 16, 2022. https://bit.ly/3Wn91Uy
