Nadofaragene firadenovec received FDA approval in December 2022 for the treatment of patients with high-risk BCG-unresponsive NMIBC with carcinoma in situ with or without papillary tumors.
The intravesical gene therapy nadofaragene firadenovec-vncg (Adstiladrin) is now fully available across the US for the treatment of adult patients with high-risk BCG-unresponsive non–muscle-invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors, according to a news release from Ferring Pharmaceuticals, the developer of the therapy.1
Nadofaragene firadenovec received FDA approval in December 2022 for use in this patient population.2 In September 2023, the ADSTILADRIN Early Experience Program was launched to give increased access to the treatment. Those included in the program were clinical trial sites that agreed to participate in the ADSTILADRIN phase 3 study, as well as to community clinics with the highest number of patients with BCG-unresponsive NMIBC CIS.
With a full supply of the treatment now ahead of schedule, the company has ended the temporary ADSTILADRIN Early Experience Program.
“From the day we received FDA approval, Ferring has been committed to making ADSTILADRIN available to every appropriate high-risk NMIBC patient quickly and responsibly, working collaboratively with the bladder cancer community to inform our approach,” said Bipin Dalmia, senior vice president and global head of the uro-oncology franchise at Ferring Pharmaceuticals, in the news release.1 “With our significant manufacturing investments, we have achieved full product supply ahead of schedule. We are therefore ending our ADSTILADRIN Early Experience Program and look forward to bringing this novel therapy to every patient who needs it.”
The phase 3 ABLE-41 US real-world evidence study (NCT06026332) of the therapy remains ongoing. The study will collect data from patients, caregivers, and prescribing physicians to assess the early utilization, experiences, and outcomes of nadofaragene firadenovec in a real-world clinical setting.
The primary outcome measure for the trial is the complete response rate, as assessed at 3 months and 1 year from first therapy instillation. Secondary outcome measures include the duration of complete response, the rate of high-grade recurrence-free survival, progression-free survival, and overall survival.
Completion of the study is expected in 2026.3
The safety and efficacy of nadofaragene firadenovec was established in the CS-003 trial (NCT02773849), which supported the FDA approval of the treatment.2 In total, the trial included 157 patients with high-risk BCG-unresponsive NMIBC CIS with or without papillary tumors. Patients received nadofaragene firadenovec once every 3 months for up to 12 months or until unacceptable toxicity or a recurrence of high-grade NMIBC.
A complete response was observed among 51% of patients who received nadofaragene firadenovec. The median duration of response was 9.7 months, and 46% of responders remained in complete response for at least 1 year.
Andrea Maddox-Smith, CEO of the Bladder Cancer Advocacy Network, concluded in the news release, “Adstiladrin represents an effective alternative therapy for patients with NMIBC who, historically, had very few options once they no longer responded to standard BCG therapy. Increasing access to this innovative therapy offers the potential of what patients need most – safe, effective treatment options that bring hope.”1
1. Ferring announces full availability of ADSTILADRIN (nadofaragene firadenovec-vncg) in the U.S. News release. Ferring Pharmaceuticals. Published online and accessed January 16, 2024. https://www.businesswire.com/news/home/20240116546395/en/Ferring-Announces-Full-Availability-of-ADSTILADRIN%C2%AE-nadofaragene-firadenovec-vncg-in-the-U.S.
2. FDA approves first gene therapy for the treatment of high-risk, non-muscle-invasive bladder cancer. News release. FDA. December 16, 2022. Accessed January 16, 2024. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treatment-high-risk-non-muscle-invasive-bladder-cancer
3. ADSTILADRIN early utilization and outcomes in the real world setting in the United States (ABLE-41). ClinicalTrials.gov. Last updated September 7, 2023. Accessed January 16, 2024. https://www.clinicaltrials.gov/study/NCT06026332