FDA approves accelerated thawing method for nadofaragene in NMIBC

The FDA has approved a label update for nadofaragene firadenovec-vncg (Adstiladrin), introducing an accelerated thawing protocol that may reduce preparation time for intravesical administration of the agent in patients with high-risk, BCG-unresponsive non–muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors, Ferring Pharmaceuticals announced.¹

The updated labeling permits thawing in a 25 °C water bath over approximately 25 minutes based on stability data demonstrating maintained product integrity under these conditions.

According to Ferring, nadofaragene is “shipped and stored as a sterile frozen suspension and must be brought to room temperature (20° C to 25° C [68° F to 77° F]) prior to use.” Thus, the accelerated thawing method may have practical implications for clinic workflow and treatment delivery in this patient population.

“The reduction in thawing time for [nadofaragene] streamlines preparation and handling for health care providers treating patients with high-risk non-muscle invasive bladder cancer,” said Vikram M. Narayan, MD, FACS, associate professor at Emory University Department of Urology and Chief of Urology at Grady Memorial Hospital, in the news release.¹ “This updated preparation process has the potential to save valuable time and enable health care providers and practices to deliver this treatment option to more patients efficiently.”

Nadofaragene firadenovec was initially approved in the US in December 2022 for adult patients with high-risk BCG-unresponsive NMIBC with carcinoma in situ (CIS), with or without papillary tumors (Ta/T1), based on results from a phase 3, single-arm trial (NCT02773849).¹ In that study, 157 patients received intravesical instillation of the gene therapy once every 3 months.

The primary efficacy end point of complete response (CR) rate in patients with CIS was achieved in 53.4% (95% CI, 43.3 to 63.3) of patients, with a median duration of response of 9.7 months (95% CI, 9.2 to 24.0). Long-term follow-up data further supported the safety and efficacy of the agent, showing that nearly half of patients remained free of cystectomy at month 60.² Treatment-related adverse events were generally manageable, with the most common including fatigue, bladder spasm, and micturition urgency.

The newly approved label update is supported by a thawing and handling study demonstrating that the frozen suspension remains stable following approximately 25 minutes of thawing in a 25 °C (77 °F) water bath. Storage conditions after thawing remain unchanged, allowing the product to be held at room temperature for up to 24 hours or refrigerated (2 to 8 °C) for up to 7 days, inclusive of thaw time.

“ADSTILADRIN already offers convenient, once-every-3-months dosing that helps reduce visit frequency, travel burden, and clinic workflow demands,” noted Denise D’Andrea, MD, FACP, Senior Director, Medical Affairs Uro-Oncology, Ferring Pharmaceuticals, in the news release. 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.”

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 24, 2026. https://www.businesswire.com/news/home/20260324850788/en/U.S.-FDA-Approves-Label-Update-to-Accelerate-Thaw-Time-for-ADSTILADRIN-nadofaragene-firadenovec-vncg

2. 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 Jul;212(1):74-86. doi:10.1097/JU.0000000000004020