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ADSTILADRIN® in Patients With High-Risk, BCG Unresponsive NMIBC

Actionable Clinical Data
Videos
Real-World Data on Nadofarogene Firadenovec for BCG-Unresponsive Bladder Cancer - Mark Tyson & Jacob Moyer
36-Month Efficacy of Nadofaragene in BCG-Unresponsive Non-Muscle-Invasive Bladder Cancer - Yair Lotan
Navigating the Expanding Landscape of BCG-Unresponsive NMIBC Treatment Options - Mark Tyson
Integrating Nadofaragene Firadenovec into Clinical Practice for Non-Muscle Invasive Bladder Cancer Treatment - Ashish Kamat
Implementing Nadofaragene Firadenovec Treatment in NMIBC - Mark Tyson, Aimee Boden, & Meghann Davis
Treatment Options Expand for BCG Unresponsive Bladder Cancer - Mark Tyson
Nadofaragene Firadenovec: Adding to the Treatment Regimen Protocol - Joshua Meeks
Predicting Recurrence and Monitoring Response in BCG-Unresponsive NMIBC with Urinary Minimal Residual Disease Detection - Vikram Narayan
Establishing Nadofaragene Firadenovec in a Large Urology Practice - Daniel Canter
Real-World Use of Nadofaragene Firadenovec - Siamak Daneshmand
Intravesical Nadofaragene Firadenovec Gene Therapy for BCG-Unresponsive NMIBC - Stephen Boorjian
Novel Gene Therapy Shows Efficacy and Tolerable Safety Profile for Patients with Treatment-Resistant Bladder Cancer - Trinity Bivalacqua
Gene Therapy for NMIBC: A New Era of Treatment - Colin Dinney
Results of the SUO CTC Phase III Nadofaragene Firadenovec Trial for BCG Unresponsive NMIBC - Colin Dinney
A Debate on The Management of BCG Unresponsive - Cystectomy Ineligible Bladder Cancer Patients: Pembrolizumab Vs. Nadofaragene Firadenovec - Arjun Balar & Peter Black
Phase III Nadofaragene Firadenovec for BCG-Unresponsive Non-Muscle Invasive Bladder Cancer - Joshua J. Meeks
Advancements in BCG Unresponsive Bladder Cancer: Highlights from the 2019 SUO Meeting - Joshua Meeks
Defining BCG-Unresponsive and The Pathway to Clinical Trials in Treating NMIBC - Ashish Kamat
Conference Coverage
Conference Highlights Written by Physician-Scientist
Presented by Amy Luckenbaugh, MD, FACS
The 2025 SESAUA annual meeting featured a bladder cancer session and a presentation by Dr. Amy Luckenbaugh discussing the durability of nadofaragene firadenovec response in participants with BCG unresponsive non-muscle invasive disease. Nadofaragene firadenovec is an intravesical gene therapy for BCG-unresponsive non–muscle invasive bladder cancer with CIS with/without papillary tumors (± Ta/T1). In a phase 3 study, 53.4% (55/103) of participants with CIS ± Ta/T1 (CIS cohort) achieved a complete response at 3 months and 72.9% (35/48) of participants with high-grade Ta/T1 (high-grade Ta/T1 cohort) were high-grade recurrence free at 3 months.1 At the 2025 SESAUA meeting, Dr. Luckenbaugh and colleagues presented results from 36 and 57 months of follow-up.
Presented by Jacob A. Moyer
The 2025 GU ASCO annual meeting featured a urothelial carcinoma session and a presentation by Jacob Moyer discussing real-world outcomes of nadofaragene firadenovec in BCG-unresponsive non-muscle invasive bladder cancer. Based on data from the 2021 key trial published in Lancet Oncology,1 nadofaragene firadenovec is an FDA-approved gene therapy for BCG unresponsive non-muscle invasive bladder cancer with promising clinical trial results. However, real-world post-marketing efficacy or safety data remain lacking. As such, Jacob Moyer and colleagues sought to evaluate complete response rates and safety in a multisite experience.
Presented by Vikram Narayan, MD
The 2024 Society of Urologic Oncology (SUO) annual meeting held in Dallas, TX between December 3rd – 6th, 2024 was host to a bladder cancer poster session. Dr. Vikram Narayan presented the results of ABLE-32, a randomized, controlled, phase IIIB clinical trial of Nadofarogene Firadenovec-vcng versus observation in patients with intermediate-risk, non-muscle invasive bladder cancer (NMIBC).
Presented by Yair Lotan, MD
The 2024 South Central AUA annual meeting included a session on bladder cancer, featuring a presentation by Dr. Yair Lotan discussing 36-month follow-up from a phase 3 trial assessing the efficacy of intravesical nadofaragene firadenovec for patients with BCG-unresponsive non muscle invasive bladder cancer (NMIBC).
Presented by Min Yang, MD, PhD
The 2024  ASCO annual meeting featured a session on bladder cancer, and a presentation by Dr. Min Yang discussing a cost effectiveness analysis of nadofaragene firadenovec for the treatment of high-risk, BCG–unresponsive, non-muscle invasive bladder cancer from a United States third-party payer perspective
Presented by Vikram Narayan, MD
The 2024 AUA annual meeting was host to a podium session presentation by Dr. Vikram Narayan who presented the final results from the 60-month follow-up of the phase 3 trial evaluating the efficacy of intravesical Nadofaragene Firadenovec-vcng for patients with Bacillus Calmette-Guerin (BCG)-unresponsive carcinoma in situ (CIS) of the bladder.
Presented by Vikram Narayan, MD
The 2024 AUA Annual Meeting was host to a non-invasive bladder cancer moderated poster session with a presentation by Dr. Vikram Narayan who presented an analysis of urinary minimal residual disease detection for predicting recurrent in BCG unresponsive non-muscle invasive bladder cancer and quantifying molecular response to nadofaragene firadenovec
Presented by Colin P. Dinney, MD
The 2024 European Association of Urology (EAU) annual meeting featured a session on non-muscle invasive bladder cancer, and a presentation by Dr. Colin Dinney discussing 36 month follow-up from the phase 3 trial assessing the efficacy of intravesical nadofaragene firadenovec-vncg for patients with BCG-unresponsive non-muscle-invasive bladder cancer.
Presented by Vikram M. Narayan, MD
The 2024 GU ASCO annual meeting featured a urothelial carcinoma session and a presentation by Dr. Vikram Narayan discussing urinary minimal residual disease detection predicting recurrence in BCG-unresponsive non-muscle-invasive bladder cancer (NIMBC) and quantifying molecular response to nadofaragene firadenovec.
Presented by Siamak Daneshmand, MD
The 2024 American Society of Clinical Oncology Genitourinary (ASCO GU) cancers symposium includes a urothelial carcinoma session featuring trials in progress and a presentation by Dr. Sia Daneshmand discussing the trial design of ABLE-41 assessing nadofaragene firadenovec-vncg early use and outcomes in a real-world setting in the United States.
Presented by Stephen Boorjian, MD
(UroToday.com) The 2023 Society of Urologic Oncology (SUO) annual meeting held in Washington, D.C. between November 28th and December 1st, 2023, was host to a poster/abstract session. Dr. Stephen Boorjian presented the 36 months follow-up results of the phase 3 trial evaluating the efficacy of intravesical nadofaragene firadenovec-vcng for patients with Bacillus Calmette-Guerin (BCG)-unresponsive carcinoma in situ (CIS) of the bladder.
Reno, Nevada (UroToday.com) -- Ferring Pharmaceuticals announced the presentation of new 36-month follow-up data from the Phase 3 study at the 24th Annual Meeting of the Society of Urologic Oncology (SUO) demonstrating a sustained durability of response with ADSTILADRIN® (nadofaragene firadenovec-vncg) in adult patients with high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors (±Ta/T1). ADSTILADRIN is the first and only intravesical gene therapy approved by the U.S. Food and Drug Administration (FDA) in this patient population.
Presented by Dr. Seth Lerner, MD
(UroToday.com) The 2022 Annual Meeting of the American Urological Association (AUA) was host to The International Bladder Cancer Group (IBCG) AUA Bladder Cancer Forum which featured a State-of-the-Art lecture by Dr. Seth Lerner regarding the current state of BCG unresponsive disease. Dr. Lerner began his talk by introducing the revised definition of “BCG Unresponsive” high risk non-muscle invasive bladder cancer (NMIBC).
Presented by Neal D. Shore, MD, FACS
(UroToday.com) As part of the “Game-changing Session 2” plenary presentation at the European Association of Urology (EAU) Virtual Annual Meeting, Dr. Neal Shore presented results of the phase III trial of nadofaregene firadenovec in patients with high-grade, bacillus calmette-guerin or BCG-unresponsive non-muscle invasive bladder cancer on behalf of Dr. Boorjian, Dr. Dinney, and the Society of Urologic Oncology (SUO) Clinical Trials Consortium (SUO CTC).
Presented by Stephen Boorjian, MD
(UroToday.com) Most patients newly diagnosed with bladder cancer have non-muscle invasive disease (NMIBC). For patients with intermediate or high-risk NMIBC and those with carcinoma in situ (CIS), adjuvant treatment with BCG is guideline-recommended on the basis of proven benefits in disease recurrence. While BCG is efficacious, many patients eventually develop BCG-unresponsive disease and are at risk for tumor recurrence and progression. 
(UroToday.com) At the AUA 2020 virtual annual meeting, Sam Chang, MD, MBA, moderated a case-based panel discussion on both non-muscle and muscle-invasive bladder cancer. Areas of focus for this discussion included (i) CIS, (ii) T1 bladder cancer, (iii) multimodality therapy for invasive disease, and (iv) advances for metastatic bladder cancer.
Presented by Wassim Kassouf, MD, FRCS
Treatment of patients with Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) remains a significant clinical challenge. At the session on Confronting Obstacles in Treating Nonmuscle-Invasive and UTUC at the 2020 ASCO GU, Dr. Wassim Kassouf presented an overview of treatment options available for these patients.
Presented by Stephen A. Boorjian, MD
San Francisco, California (UroToday.com) For patients with BCG unresponsive non-muscle invasive bladder cancer, the standard of care for patients who are operative candidates is radical cystectomy. However, not all patients may be cystectomy candidates, often for a multitude of reasons, including coexisting comorbidities as well as personal considerations and quality of life.1
Presented by Colin Dinney, MD
Washington, DC (UroToday.com) During the bladder cancer session at the Society of Urologic Oncology Meeting on Thursday, December 5th, Dr. Colin Dinney presented the results of a phase III clinical trial for Adstiladrin® (rAd-INFa/syn3) in BCG unresponsive non-muscle invasive bladder cancer.
San Francisco, CA (UroToday.com) -- FerGene, a new gene therapy company formed by Ferring Pharmaceuticals and Blackstone Life Sciences, announced positive results from the pivotal Phase 3 clinical trial evaluating nadofaragene firadenovec (rAd-IFN/Syn3), an investigational gene therapy, for the treatment of high-grade, Bacillus Calmette-Guérin (BCG) unresponsive non-muscle invasive bladder cancer (NMIBC). 
Presented by Badrinath Konety, MD, MBA
Athens, Greece (Urotoday.com) Dr. Badrinath Konety presented on intravesical therapy for non-muscle invasive bladder cancer (NMIBC). The current risk-based therapy entails:
Presented by Stephen Boorjian, MD
Chicago, IL (UroToday.com) Dr. Steve Boorjian from the Mayo Clinic provided a high-level talk discussing the clinical perspective of BCG unresponsiveness in non-muscle-invasive bladder cancer (NMIBC) at the SBUR session at AUA 2019. Dr. Boorjian notes that defining patients with BCG unresponsive disease means that these are the patients we need to identify that should not receive further BCG.
Presented by Colin Dinney, MD
Chicago, IL (UroToday.com) In a podium presentation as part of the Society of Urologic Oncology program at the American Urologic Association Annual Meeting, Dr. Colin Dinney presented the late-breaking phase 2 data regarding Nadofaragene firadenovec (Adstiladrin®) (rAd-interferon-alpha/Syn3; nadofaragene firadenovec) in BCG unresponsive non-muscle invasive bladder cancer (NMIBC). The twelve-month data from this trial was previously reported and published in the Journal of Clinical Oncology. At twelve months, 35% of patients were free of high-grade recurrence.
Publications
Peer-Reviewed Journal Abstracts
  • Patients with high-risk, non-muscle invasive bladder cancer now have greater access to the first and only FDA-approved intravesical gene therapy
  • With full supply of ADSTILADRIN ahead of schedule, Ferring ends the temporary ADSTILADRIN Early Experience Program
  • Enrollment in ABLE-41 U.S. Real-World Evidence Study is ongoing

Bladder cancer is a prevalent malignancy with limited therapeutic options, particularly for patients who are unresponsive to Bacillus Calmette-Guérin (BCG). The approval of interferon-α (IFNα) gene therapy with nadofaragene firadenovec (Adstiladrin®), the first gene therapy for genitourinary malignancies, has provided a promising alternative.

This article reviews the research and milestones that led to the development and approval of nadofaragene firadenovec. Bladder cancer is well-suited for gene therapy due to direct access to the bladder and the availability of urine and tissue samples for monitoring.

  • Ferring’s novel adenovirus vector-based gene therapy Adstiladrin® (nadofaragene firadenovec-vncg) is the first gene therapy approved for bladder cancer
  • Efficacy and safety of Adstiladrin supported by Phase 3 results demonstrating that more than half of patients (51% of CIS ± Ta/T1 cohort) achieved a complete response (CR) at three months and of these, 46% continued to remain free of high-grade recurrence at 12 months
  • Bladder cancer is the sixth most common cancer in the U.S.; Adstiladrin provides NMIBC patients a valuable alternative compared to an invasive bladder removal surgery

A newly published systematic review and meta-analysis: Evidence-based Assessment of Current and Emerging Bladder-sparing Therapies for Non–muscle-invasive Bladder Cancer After Bacillus Calmette-Guerin Therapy: A Systematic Review and Meta-analysis 

Durable responses in 35 percent of patients reported in Phase II data published in the Journal of Clinical Oncology

Truckee, CA., [August 27, 2017] – The Society of Urologic Oncology and the Society of Urologic Oncology Clinical Trials Consortium (SUO-CTC) announced that the Journal of Clinical Oncology (JCO) has published results from a Phase II clinical study led by the SUO- CTC demonstrating the potential effectiveness of Nadofaragene firadenovec (Adstiladrin®) (rAD-IFN/Syn3) in patients with high grade, BCG refractory or relapsed Non-Muscle Invasive Bladder Cancer (NMIBC).