FDA Approves First Gene Therapy for Bladder Cancer

On December 16, the FDA approved Adstiladrin (nadofaragene firadenovec-vncg), a non-replicating adenoviral vector-based gene therapy for the treatment of adult patients with high-risk, non-muscle-invasive bladder cancer (NMIBC). It’s the first FDA-approved gene therapy for bladder cancer. 

More specifically, Adstiladrin is indicated for high-risk Bacillus Calmette-Guérin immunotherapy-unresponsive NMIBC with carcinoma in situ (CIS) with or without papillary tumors. It’s administered every three months directly into the bladder through a catheter. 

Bladder cancer is one of the most common forms of cancer. The American Cancer Society estimated 81,180 new cases in 2022. It’s the fourth most common cancer in men. NMIBC (aka superficial) bladder cancers are ones that haven’t grown into the muscle layer. They make up most of the newly diagnosed cases and include non-invasive (stage 0) and some early (stage 1) invasive tumors. 

Standard treatment for NMIBC currently involves removing the tumor and then using Bacillus Calmette-Guérin immunotherapy to reduce the risk of recurrence. For people resistant to this treatment, few effective options exist.

The FDA approval came after results of an open-label, single-arm Phase 3 study. Participants received repeat doses of Adstiladrin at months 3, 6, and 9. Overall, 51% of enrolled patients with CIS using Adstiladrin therapy achieved a complete response. Forty-six percent of them remained in complete response for at least a year. Patients will continue to be monitored for a total of five years. 

The results of the Adstiladrin study were published in November 2020 in The Lancet Oncology. The FDA granted approval of Adstiladrin to Ferring Pharmaceuticals A/S. The therapy is expected to become commercially available in the second half of 2023.

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