FDA Approves First Gene Therapy for Specific Form of Bladder Cancer

The FDA has approved Ferring’s gene therapy Adstiladrin to treat adults with high-risk Bacillus Calmette-Guérin-unresponsive non-muscle-invasive bladder cancer — the first gene therapy approved for this patient population.

Bacillus Calmette-Guérin immunotherapy is still the standard of care as first-line treatment in patients with high-grade non-muscle invasive bladder cancer, but many patients become unresponsive to the treatment.

Adstiladrin (nadofaragene firadenovec-vncg) is administered through a catheter into the bladder once every three months. The FDA approval was supported by results from a clinical trial in which 51 percent of participants achieved a complete response and 46 percent of responding patients sustained the response for at least one year.

Ferring’s application received Breakthrough Therapy and Fast Track designations as well as a Priority Review.