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Adstiladrin is an adenoviral vector-based gene therapy that is non-replicating, meaning it cannot multiply in human cells. Its safety and effectiveness were evaluated in a multicentre clinical study, which included 157 patients with high-risk BCG-unresponsive NMIBC, of which 98 had BCG-unresponsive CIS with or without papillary tumours and could be evaluated for response.

Patients received Adstiladrin once every three months for up to 12 months, or until there was unacceptable toxicity to therapy or recurrent high-grade NMIBC. The study found that, overall, 51% of enrolled patients achieved a complete response: the disappearance of all signs of cancer as seen on cystoscopy, biopsied tissue and urine. The median duration of response was 9.7 months, with 46% of the responding patients remaining in complete response for at least one year.

Peter Marks MD PhD, director of the FDA's Center for Biologics Evaluation and Research stated: “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. 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.”