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The FDA nadofaragene firadenovec (Adstiladrin) as the first gene therapy for the treatment of high-risk bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer (NMIBC), the agency announced on Friday.

An adenoviral vector-based product, nadofaragene firadenovec is specifically indicated for adult patients with carcinoma in situ (CIS) with or without papillary tumors.

Approval was based on results from a multicenter clinical study that included 157 patients with high-risk BCG-unresponsive NMIBC, 98 of whom had BCG-unresponsive CIS with or without papillary tumors and could be evaluated for response.

"Patients with BCG-unresponsive NMIBC have historically had limited treatment options other than bladder removal surgery," said lead investigator Stephen Boorjian, MD, of the Mayo Clinic in Rochester, Minnesota, in a . This approval "is therefore a significant advance in the current treatment landscape and provides a novel treatment option for patients."

In the study, patients received nadofaragene firadenovec once every 3 months for up to 12 months, or until unacceptable toxicity to therapy or recurrent high-grade NMIBC. The complete response (CR) rate was 51% at 3 months, with about half of these responding patients remaining in CR for at least 1 year. The median duration of response was 9.7 months.

"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," said Peter Marks, MD, PhD, director of the FDA's Center for Biologics Evaluation and Research, in a statement. "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."

Nadofaragene firadenovec is a non-replicating adenovirus vector-based therapy containing the interferon alfa-2b gene. As described by developer Ferring Pharmaceuticals, the vector enters the cells of the bladder wall and releases the interferon alfa-2b gene.

"The internal gene/DNA machinery of the cells 'picks up' the gene and translates its DNA sequence, resulting in the cells secreting high quantities of interferon alfa-2b protein, a naturally occurring protein the body uses to fight cancer," according to the company.

The product is administered once every 3 months into the bladder via a urinary catheter. The most common adverse events associated with nadofaragene firadenovec included bladder discharge, fatigue, bladder spasm, urinary urgency, hematuria, chills, fever, and painful urination.

The gene therapy is not indicated for individuals who are immunosuppressed or immune-deficient.

Ferring expects nadofaragene firadenovec to be commercially available in the U.S. in the second half of 2023.

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