Nadofaragene Firadenovec Delivers Durable Responses in Bladder Cancer

Data from a 3-year follow-up analysis of a phase 3 study (NCT02773849) evaluating nadofaragene firadenovec-vncg (Adstiladrin) in high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) showed that the agent delivered a sustained, durable response.^1,2

The data were presented at the 39th Annual European Association of Urology Congress and showed that in the carcinoma in situ (CIS) cohort, 53% of patients achieved a complete response (CR) at month 3, and 25.5% of these patients remained high-grade recurrence-free by 36 months. In the high-grade papillary disease cohort, almost 73% of patients were high-grade recurrence-free at month 3, and about 31% of these patients remained recurrence-free through 3 years.

“[Nadofaragene firadenovec] is a novel therapy that has demonstrated its value as an effective and well-tolerated standard-of-care treatment for high-risk NMIBC patients with CIS ± Ta/T1 who have BCG-unresponsive disease,” said Pierre-Yves Berclaz, MD, PhD,executive vice president and chief science and medical officer, Ferring Pharmaceuticals, in a press release.¹ “This 3-year analysis provides further evidence for the durable efficacy and long-term safety of [nadofaragene firadenovec] in this on-label patient population, as well as additional data showing its therapeutic potential in a separate population of NMIBC patients with papillary disease.”

In both cohorts, the median duration of CR was 10 months (range, 9.2-24.0), and the Kaplan-Meier estimated probability of duration of CR for at least 3 years was 34.2% (range, 21.6%-47.1%). The estimated median duration of high-grade recurrence-free survival was 6 months (95% CI, 3.4-8.3) in the CIS cohort and about 12 months (95% CI, 6.7-20.3) in the papillary disease cohort.^1,2

Estimated cystectomy-free survival at 3 years was about 54% (95% CI, 43.3%-63.1%) in the CIS arm vs almost 64% (95% CI, 48.0%-75.6%) in the papillary disease arm. Three-year overall survival was about 90% (range, 82.3%-94.9%) and 91% (range, 78.5%-96.7%) in the CIS and papillary disease cohorts, respectively.^1,2

No new safety signals were identified during the 3-year follow-up, and only 3 patients discontinued treatment due to an adverse event. These included grade 3 bladder spasm, grade 2 instillation site discharge, and grade 2 benign bladder neoplasm.

In December 2022, the FDA approved nadofaragene firadenovec in this intent-to-treat population,³ and the agent was made fully available in the US in January 2024.⁴

“We look forward to continuing patient follow up as we work to redefine the treatment of NMIBC,” added Berclaz, in the press release.

REFERENCES:

1. Boorjian SA, Narayan VM, Konety BR, et al. Efficacy of intravesical nadofaragenefiradenovec-vncg for patients with Bacillus Calmette-Guérin-unresponsive non-muscle-invasive bladder cancer: 36-month follow-up from a phase 3 trial. Presented at the 39th Annual European Association of Urology Congress. Paris, France. April 5-8, 2024. Abstract A0583

2. Three-year follow-up data in BCG-unresponsive NMIBC show durable response to treatment with ADSTILADRIN® (nadofaragenefiradenovec-vncg) in two patient cohorts. News release. Ferring Pharmaceuticals. April 8, 2024. Accessed April 8, 2024. https://tinyurl.com/5e6558cp 

3. Ferring receives approval from U.S. FDA for Adstiladrin for high-risk, BCG-unresponsive non-muscle invasive bladder cancer. News release. Ferring Pharmaceuticals. December 16, 2022. Accessed April 8, 2024. https://bit.ly/3YxyWdY 

4. Ferring announces full availability of ADSTILADRIN® (nadofaragene firadenovec-vncg) in the U.S. News release. Ferring Pharmaceuticals. January 16, 2024. Accessed April 8, 2024. http://tinyurl.com/7dy6nmd9