FDA Approves First Gene Therapy for Non-Muscle Invasive Bladder Cancer

Patients with high-risk Bacillus Calmette Guérin–unresponsive non-muscle invasive bladder cancer can now receive treatment with nadofaragene firadenovec-vncg—the first gene therapy—following its approval by the FDA.

The FDA has approved nadofaragene firadenovec-vncg (Adstiladrin) for the treatment of patients high-risk Bacillus Calmette Guérin (BCG)–unresponsive non-muscle invasive bladder cancer with carcinoma in-situ plus or minus papillary tumors, according to a press release from the agency.

The non-replicating adenoviral vector–based gene therapy was assessed as part of a multicenter clinical trial in a population of 157 patients, of whom 98 had unresponsive BCG carcinoma in-situ with or without papillary tumors. Treatment with nadofaragene firadenovec was administered once every 3 months until 12 months, unacceptable toxicity, or recurrent disease.

The use of study drug resulted in a complete response (CR) rate of 51%. The median duration of response was 9.7 months, with 46% of patients maintaining their response up to 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, said in a press release. “Today’s action addresses an area of critical need.”

Study drug is administered through a urinary catheter into the bladder. The most common treatment-related adverse effects included, but were not limited to, fatigue, painful urination and hematuria.


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/3htRnzJ

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