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

Article

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.

Reference

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

Related Videos
Considering cystectomy in patients with bladder cancer may help with managing the shortage of Bacillus Calmette-Guerin, according to Joshua J. Meeks, MD, PhD, BS.
Patients with locally advanced or metastatic urothelial cancer and visceral disease may particularly benefit from enfortumab vedotin plus pembrolizumab, according to Amanda Nizam, MD.
Cretostimogene grenadenorepvec’s efficacy compares favorably with the current nonsurgical standards of care in high-risk, Bacillus Calmette Guerin–unresponsive non-muscle invasive bladder cancer.
Experts on bladder cancer
Experts on bladder cancer
Experts on bladder cancer
Experts on bladder cancer
Experts on bladder cancer
Experts on bladder cancer
Experts on bladder cancer
Related Content