Bladder Cancer

FDA Approves First Gene Therapy to Treat High-Risk Form of Bladder Cancer

Jessica Ganga

The FDA has approved the first gene therapy—nadofaragene firadenovec-vncg (Adstiladrin)—for the treatment of high-risk non-muscle-invasive bladder cancer (NMIBC) in adult patients who are unresponsive to Bascillus Calmette-Guérin (BCG) with carcinoma in situ (CIS) with or without papillary tumors.

“Today’s action addresses an area of critical need. The FDA remains committed to facilitating the development and approval of safe and effective cancer treatments,” said Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, in a press release.

NMIBC is a common diagnosis for those newly diagnosed with bladder cancer (75% to 80%) and is associated with high rates of recurrence (between 30% to 80%) along with the risk of disease progression to invasive and metastatic cancer.

The approval follows a multicenter clinical trial study of patients with high-risk BCG-unresponsive NMBIC. In total, 157 patients were included, 98 of whom had BCG-unresponsive CIS with or without papillary tumors. Patients received treatment once every 3 months for up to 12 months. The researchers found that 51% of patients who used the gene therapy achieved complete response—defined as the disappearance of all signs of cancer. The median duration of response was 9.7 months.

The gene therapy is administered to patients once every 3 months in the bladder using a urinary catheter. The most common adverse events include bladder discharge, fatigue, bladder spasm, urinary urgency, hematuria, chills, fever, and painful urination. Further, patients who are immunosuppressed or immune-deficient are not recommended for treatment.



FDA approves first gene therapy for the treatment of high-risk, non-muscle-invasive bladder cancer. News release. US Food and Drug Administration; December 16, 2022. Accessed January 4, 2023.