The U.S. Food and Drug Administration (FDA) has granted approval to Ferring Pharmaceuticals A/S for a new gene therapy to treat bladder cancer. The Dec. 16, 2022, announcement stated that the approval of Adstiladrin (nadofaragene firadenovec-vncg) would provide a new treatment option for adults who were not responsive to more traditional treatments.

According to the CDC, approximately 57,000 men and 18,000 women are diagnosed with bladder cancer annually, and roughly 12,000 men and 4,700 women die from the disease each year in the United States. 

When a patient has bladder cancer, abnormal cells invade and destroy bladder tissue. 75-80% of bladder cancers are classified as non-muscle-invasive, meaning that the bladder cancer has grown through the lining of the bladder but has not metastasized to the surrounding muscle. However, this type of cancer has a recurrence rate of up to 80% and is at risk of progressing to invasive and metastatic cancer.

Treatment of patients with non-muscle-invasive bladder cancer that features cancer cells in the location where they first formed without spreading to nearby tissue (carcinoma in situ) involves the removal of the tumor and the implementation of Bacillus Calmette-Guérin (BCG) therapy. BCG therapy delivers inactive tuberculosis bacteria through a catheter inserted into the urethra in order to generate an inflammatory response and prevent the cancer from returning.

The FDA announcement states that few effective treatment options exist for patients who develop BCG-unresponsive bladder cancer. The failure of BCG therapy or the disappearance of all signs of the cancer is associated with an increased risk of death or an intensification of the cancer.

Adstiladrin is a non-replicating adenoviral vector-based gene therapy. This means that it cannot multiply in human cells and spreads using a deactivated adenovirus. Adstiladrin will be used for non-muscle-invasive bladder cancer unresponsive to BCG therapy. In clinical trials, Adstiladrin helped 51% of the 157 enrolled patients achieve a complete disappearance of the cancer, with 46% keeping bladder cancer from recurring for at least a full year.

Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research, stated in the announcement that Adstiladrin’s approval “addresses an area of critical need.” Furthermore, he reaffirmed the agency’s commitment to “facilitating the development and approval of safe and effective cancer treatments.”