FDA Approves Label Update for Nadofaragene Firadenovec in NMIBC

The FDA has approved a label update to nadofaragene firadenovec-vncg (Adstiladrin) that introduces an accelerated water bath thawing method completed in approximately 25 minutes, a change intended to streamline clinical preparation for health care teams managing patients with high risk non–muscle-invasive bladder cancer (NMIBC), according to a manufacturer news release.¹

Nadofaragene firadenovec is the first and only FDA approved nonreplicating intravesical gene therapy for the treatment of adult patients with high risk Bacillus Calmette Guérin unresponsive NMIBC with carcinoma in situ with or without papillary tumors. Bladder cancer is the sixth most common cancer in the United States, with NMIBC accounting for about 75% of all new cases.

“The reduction in thawing time for Adstiladrin streamlines preparation and handling for healthcare providers treating patients with high-risk non-muscle invasive bladder cancer,” Vikram M. Narayan, MD, FACS, associate professor at Emory University and chief of urology at Grady Memorial Hospital, said in a release. “This updated preparation process has the potential to save valuable time and enable healthcare providers and practices to deliver this treatment option to more patients efficiently.”

Nadofaragene firadenovec is shipped and stored as a sterile frozen suspension and must be brought to room temperature prior to use. The approval was supported by a thawing and handling study demonstrating that frozen vials remained stable when thawed for about 25 minutes in a water bath maintained at 25 degrees Celsius. The storage conditions remain unchanged once thawing begins and permit storage for up to 24 hours at room temperature or up to 7 days refrigerated, including thaw time.

“At Ferring, we remain deeply committed to evolving our therapies in ways that address the real-world needs of patients and the healthcare providers who care for them,” Denise D’Andrea, MD, FACP, senior director of Medical Affairs Uro Oncology at Ferring Pharmaceuticals, said in a release. She noted that nadofaragene firadenovec offers convenient once every 3 months dosing that helps reduce visit frequency, travel burden, and clinic workflow demands, and the new thaw option provides additional flexibility to streamline operational workflow across care settings.

For patients with high risk NMIBC, intravesical BCG remains the first line standard of care, but approximately one third of patients will not respond to BCG therapy, and 50% of those with an initial response will experience recurrence or progression. Current treatment options for BCG unresponsive patients are limited, and national guidelines recommend cystectomy.

The FDA approved the agent in December 2022 based on efficacy results from Study CS-003 (NCT02773849), a single-arm trial that enrolled 157 patients with high-risk NIMBC.² The major efficacy outcome measures were complete response at any time and duration of response. Complete response was defined as negative cystoscopy with applicable transurethral resection of bladder tumor and biopsies and urine cytology. Random bladder biopsies of five sites were conducted in patients remaining in complete response at 12 months. The complete response rate was 51%, the median duration of response was 9.7 months, and 46% of responding patients remained in complete response for at least one year.

Nadofaragene firadenovec is a non-replicating adenovirus vector based therapy containing the gene interferon alfa 2b, administered locally as a monotherapy by catheter directly into the bladder once every three months. The vector enters the cells of the bladder wall, releasing the active gene and causing the bladder’s cell walls to secrete high and transient local expression of interferon alfa 2b protein, a naturally occurring protein the body uses to fight cancer. This approach essentially turns the bladder wall cells into interferon microfactories, enhancing the body’s own natural defenses against the cancer.

REFERENCES

1. US FDA Approves Label Update to Accelerate Thaw Time for Adstiladrin (nadofaragene firadenovec-vncg). News release. March 24, 2026. Accessed March 25, 2026. https://tinyurl.com/4wed7z4w

2. Colbert L, Jia Y, Sharma A, et al. FDA Approval Summary: Nadofaragene Firadenovec-vncg for Bacillus Calmette-Guérin-Unresponsive Non-Muscle-Invasive Bladder Cancer. Clin Cancer Res. 2025;31(7):1182-1185. doi:10.1158/1078-0432.CCR-24-2812