Nadofaragene Firadenovec Label Update Approved in BCG-Unresponsive NMIBC

The FDA has approved a label update for nadofaragene firadenovec-vncg (Adstiladrin) to enhance efficient clinical preparation for healthcare teams, clearing the way for an accelerated 25-minute water-bath thawing method, according to a news release from the developer, Ferring Pharmaceuticals.¹

According to the release, nadofaragene firadenovec, a gene therapy, is typically shipped and stored in a sterile frozen suspension before being brought to room temperature—approximately 68° F to 77° F or 20° C to 25° C—prior to use. The approval is supported by a study that demonstrated that frozen vials remained stable when thawed in a water bath maintained at 77° F or 25° C. Moreover, the storage conditions for the gene therapy remain unchanged upon initiation of thawing, with storage permitted for up to 24 hours at room temperature or 7 days while refrigerated at 2° C to 8° C.

Nadofaragene firadenovec received FDA approval for the treatment of patients with high-risk Bacillus Calmette Guérin (BCG)–unresponsive non–muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) plus or minus papillary tumors in December 2022.²

“The reduction in thawing time for [nadofaragene firadenovec] streamlines preparation and handling for healthcare providers treating patients with high-risk [NMIBC],” Vikram M. Narayan, MD, FACS, associate professor at Emory University Department of Urology and chief of Urology at Grady Memorial Hospital, said in the news release.¹ “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.”

According to 5-year follow-up analysis of a phase 3 trial (NCT02773849) evaluating the gene therapy in patients with BCG-unresponsive non–muscle invasive bladder cancer with or without CIS, the cystectomy-free survival was 49% (95% CI, 40.0%-57.1%) among all patients enrolled (n = 151) at 60 months, including 59% (95% CI, 43.1%-71.4%) of those in the high-grade Ta or T1 cohorts and 43% (95% CI, 32.2%-53.7%) of those in the CIS cohort.³ Additionally, 59.6% (95% CI, 51.3%-67.5%) of all patients experienced a complete response (CR) or high-grade recurrence-free (HGRF) survival at 3 months of follow-up, including 72.9% (95% CI, 58.2%-84.7%) of the Ta or T1 cohort and 53.4% (95% CI, 43.4%-63.3%) of the CIS cohort.

Furthermore, the median duration of HGRF survival was 7.3 months (95% CI, 5.7-11.9), 12.4 months (95% CI, 6.7-20.3), and 5.9 months (95% CI, 3.4-8.3) in the overall, Ta or T1, and CIS populations, respectively. A total of 8.6% (95% CI, 4.7%-14.3%), 15% (95% CI, 6.1%-27.8%), and 5.8% (95% CI, 2.2%-12.2%) of the respective groups were free from high-grade recurrence at month 57 of the study.

“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, concluded in the news release.¹ “[Nadofaragene firadenovec] already offers convenient, once-every-3-months dosing that helps reduce visit frequency, travel burden and clinic workflow demands. The new 25 minutes at 25° C thaw option provides additional flexibility and represents another important step in our ongoing efforts to streamline operational workflow across care settings.”

Nadofaragene firadenovec is contraindicated among those with hypersensitivity reactions to interferon alfa or to any component of the product. Additionally, the warning and precautions list a risk with delayed cystectomy, specifically for patients with CIS, who are advised to consider cystectomy if a CR is not attained after 3 months of treatment or in the event of CIS recurrence. Additionally, the risk of disseminated infection may be higher among those who are immunocompromised or immunodeficient, and nadofaragene firadenovec is not advised for these patients.

References

1. U.S. FDA approves label update to accelerate thaw time for ADSTILADRIN® (nadofaragene firadenovec-vncg). News release. Ferring Pharmaceuticals. March 24, 2026. Accessed March 25, 2026. https://tinyurl.com/4cn3haua
2. FDA approves first gene therapy for the treatment of high-risk, non-muscle-invasive bladder cancer. News release. FDA. December 16, 2022. Accessed March 25, 2026. https://tinyurl.com/4j6nj4cz
3. Narayan VM, Boorjian SA, Alemozaffar M, et al. Efficacy of intravesical nadofaragene firadenovec for patients with Bacillus Calmette-Guérin–unresponsive nonmuscle-invasive bladder cancer: 5-year follow-up from a phase 3 trial. J Urol. 2024;212(1):74-86. doi:10.1097/JU.0000000000004020