Nadofaragene Firadenovec Produces Sustained, Durable Responses in NMIBC

Nadofaragene Firadenovec Produces Sustained, Durable Responses in NMIBC

Nadofaragene firadenovec-vncg (Adstiladrin) delivered a sustained, durable response in patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC), as demonstrated by data from a 3-year follow-up analysis of a phase 3 study (NCT02773849).^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