Adstiladrin (nadofaragene firadenovec-vncg) / Ferring, FKD Therapies, Royalty - LARVOL DELTA

On 26 March 2026, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a conditional marketing authorisation for the medicinal product Adstiladrin. (European Medicines Agency) - "As Adstiladrin is an advanced therapy medicinal product, the CHMP positive opinion is based on an assessment by the Committee for Advanced Therapies....Adstiladrin is indicated as monotherapy for the treatment of adult patients with Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumours."

CHMP • Bladder Cancer

U.S. FDA Approves Label Update to Accelerate Thaw Time for ADSTILADRIN (nadofaragene firadenovec-vncg) (Businesswire) - "Accelerated water-bath thawing, completed in about 25 minutes, may help streamline clinical workflows and support efficient patient management. Label update includes data showing that product stability is maintained with the 25-minute, 25° C water-bath method."

FDA event • Bladder Cancer

LUNAR: Safety and efficacy evaluation of nadofaragene firadenovec instilled into the renal pelvis in subjects with low-grade upper tract urothelial carcinoma—A single-arm, open-label phase 1/2 trial. (ASCO-GU 2026) - P1/2 | "Secondary efficacy endpoints include duration of response and urinary excretion of IFN-α2b protein. Results from this trial are expected in 2029."

Clinical • P1/2 data • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • Urothelial Cancer • IFNA1

Indirect Treatment Comparison of Nogapendekin Alfa Inbakicept-pmln plus Bacillus Calmette–Guérin (NAI+BCG) and Nadofaragene Firadenovec-vncg in patients with BCG-unresponsive Non-Muscle Invasive Bladder Cancer CIS ± Papillary (NMIBC). (AUA 2026) - No abstract available

Clinical • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

005IC - Bladder Cancer in the Office from Indolent to Aggressive: A Practical Guideline-based Approach (AUA 2026) - "With the proliferation of options (e.g. Adstiladrin, Anktiva, gemcitabine/docetaxel, pembrolizumab), it is critical to understand 1) indications for these agents, 2) timing and sequencing of administration, and 3) management of common side effects...New systemic therapies have emerged (e.g. enfortumab vedotin, pembrolizumab, erdafitinib) and urology providers need to be aware of the changing landscape of advanced disease...Learning Objective 7: Identify patients that are candidates for new systemic therapies for advanced bladder cancer, including the FDA-approved indications. Learning Objective 8: Identify the common side effects and management strategies of new systemic therapies for advanced bladder cancer."

Bladder Cancer • Genito-urinary Cancer • Oncology • Palliative care • Solid Tumor • Urology

Advances in Intravesical Therapy for Non-Muscle-Invasive Bladder Cancer. (PubMed, Crit Rev Oncol Hematol) - "Intermediate-risk patients could benefit from either intravesical chemotherapy or Bacillus Calmette-Guérin (BCG) instillation, with emerging therapies such as UGN-102 chemoablation showing promise...Moreover, alternative agents such as sequential gemcitabine and docetaxel instillation have demonstrated favorable outcomes in both BCG-naïve and BCG-unresponsive patients...Gene therapy (nadofaragene firadenovec), oncolytic viral therapy (CG0070), interleukin-15 superagonist (Nogapendekin alfa-inbakicept), and innovative drug delivery systems (TAR-200) have shown encouraging clinical potential in this population. This review provides an overview of current intravesical treatment strategies for NMIBC across different risk groups and highlights promising new approaches aimed at overcoming the limitations of BCG therapy."

Journal • Review • Bladder Cancer • Gene Therapies • Genito-urinary Cancer • Oncology • Solid Tumor • IL15

Ferring Announces Update to NCCN Guidelines Expanding Category 2A Recommendation for Nadofaragene Firadenovec-vncg (ADSTILADRIN) in Patients with BCG-Unresponsive NMIBC (Businesswire) - "Nadofaragene firadenovec-vncg received an NCCN Category 2A recommendation for patients with papillary tumors (+Ta/T1) without carcinoma in situ (CIS), an upgrade from its 2B recommendation. Nadofaragene firadenovec-vncg maintained its Category 2A recommendation for patients with CIS with or without papillary tumors (+Ta/T1)."

NCCN guideline • Bladder Cancer

ABLE-32: Trial of Nadofaragene Firadenovec vs. Observation in Participants With Intermediate Risk Non-Muscle Invasive Bladder Cancer (clinicaltrials.gov) - P3 | N=454 | Recruiting | Sponsor: Ferring Pharmaceuticals | N=296 ➔ 454

Enrollment change • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

000434: A study to learn about nadofaragene firadenovec given alone or together with gemcitabine and docetaxel or, pembrolizumab in adults with non-muscle invasive bladder cancer and no response to treatment with Bacillus Calmette-Guerin (clinicaltrialsregister.eu) - P2/3 | N=143 | Recruiting | Sponsor: Ferring Pharmaceuticals A/S

New P2/3 trial • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

Neoadjuvant Intravesical Nadofaragene Firadenovec With Gemcitabine, Cisplatin and Durvalumab for the Treatment of Muscle Invasive Bladder Cancer, TRIFECTA Trial (clinicaltrials.gov) - P2 | N=33 | Not yet recruiting | Sponsor: University of Washington | Initiation date: Mar 2026 ➔ Jun 2026

Trial initiation date • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • Urothelial Cancer

A Real-World Case Series of Re-Induction With Nadofaragene Firadenovec in Patients With Carcinoma In Situ ± Papillary Non–Muscle-Invasive Bladder Cancer (AUA 2026) - No abstract available

Clinical • Real-world • Real-world evidence • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

Diuretic Effect Associated with Nadofaragene Firadenovec Instillation Therapy - Insight into Treatment Tolerability (AUA 2026) - No abstract available

Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • Urothelial Cancer

Clinical Outcomes of Nadofaragene Firadenovac in BCG Unresponsive NMIBC: A Multi-Center analysis (AUA 2026) - No abstract available

Clinical • Clinical data • Bladder Cancer

Efficacy of Nadofaragene firadenovec in Doubly Resistant Patients: A Real World Case Series (AUA 2026) - No abstract available

Clinical • Real-world • Real-world evidence • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • Urothelial Cancer

Budget impact model of nadofaragene firadenovec for the treatment of high-risk non-muscle-invasive bladder cancer that is unresponsive to Bacillus Calmette-Guérin immunotherapy. (PubMed, J Med Econ) - "Key limitations include reliance on projected market shares for nadofaragene firadenovec, omission of recently approved treatments (nogapendekin alfa inbakicept-pmln and TAR-200) due to the lack of publicly available market share data, and assumptions to derive clinical outcomes for certain treatments due to lack of reported data. Despite its higher acquisition cost, the inclusion of nadofaragene firadenovec resulted in a minimal budget impact as bladder-sparing option for patients with BCG-unresponsive NMIBC with CIS ± Ta/T1."

HEOR • Journal • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

000425: Low-grade UTUC Treated With Nadofaragene Firadenovec Administered to the Renal Pelvis (LUNAR) (clinicaltrialsregister.eu) - P1/2 | N=5 | Not yet recruiting | Sponsor: Ferring Pharmaceuticals A/S

New P1/2 trial • Oncology • Solid Tumor • Urothelial Cancer

Recent Advances in Immunotherapy for Non-Muscle-Invasive Bladder Cancer. (PubMed, Cancers (Basel)) - "Over the past decade, investigational trials in immunotherapy have expanded treatment options for BCG-unresponsive NMIBC with CIS, leading to FDA approval of intravesical nadofaragene firadenovec, nogapendekin alfa-inbakicept, and systemic pembrolizumab. This narrative review summarizes developments in intravesical and systemic immunotherapies for NMIBC, highlights ongoing trials, and addresses controversies in trial design, treatment sequencing, comparative efficacy, and safety."

Journal • Review • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • Urethral Cancer

ABLE-41: ADSTILADRIN Early Utilization and Outcomes in the Real World Setting (clinicaltrials.gov) - P=N/A | N=202 | Terminated | Sponsor: Ferring Pharmaceuticals | N=400 ➔ 202 | Trial completion date: Dec 2027 ➔ Jan 2026 | Recruiting ➔ Terminated | Trial primary completion date: Dec 2027 ➔ Jan 2026; An internal audit identified major inconsistencies and missing data in key registry fields, including primary outcomes. These issues mean we can no longer ensure the validity of the primary endpoints.

Enrollment change • Real-world evidence • Trial completion date • Trial primary completion date • Trial termination • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

UPDATED EFFICACY AND SAFETY OF ORAL ERDAFITINIB IN PATIENTS WITH BACILLUS CALMETTE-GUÉRIN–UNRESPONSIVE, HIGH-RISK NON-MUSCLE–INVASIVE BLADDER CANCER WITH FGFR3/2 ALTERATIONS IN THOR-2 COHORT 2 (SUO 2023) - P2 | "Treatment options for patients with bacillus Calmette-Guérin (BCG)-unresponsive CIS who refuse/are ineligible for radical cystectomy include pembrolizumab, intravesical chemotherapy, or nadofaragene firadenovec. Data from this trial provide first evidence of occurrence of FGFR alterations in CIS and demonstrate the promising and durable efficacy of an FGFR inhibitor such as erdafitinib in patients with BCG-unresponsive CIS with FGFR alterations. Safety data were consistent with the known safety profile of erdafitinib."

Clinical • Acute Kidney Injury • Bladder Cancer • Chronic Kidney Disease • Dental Disorders • Genito-urinary Cancer • Hypotension • Infectious Disease • Metabolic Disorders • Nephrology • Oncology • Renal Disease • Retinal Disorders • Septic Shock • Solid Tumor • Stomatitis • Urothelial Cancer • Xerostomia • FGFR3

Current use of pre-clinical models for evaluation of human adenovirus-based cancer therapeutics. (PubMed, Mol Ther Oncol) - "Human adenoviruses (HAdVs) are enjoying a renaissance in interest for the development of cancer therapies following the approval of Adstiladrin (Ferring Pharmaceuticals) for the treatment of bladder cancer, strong phase 3 data from CG Oncology's cretostimogene grenadenorepvec, and many other products in late-stage clinical development. These modifications, and the highly complex interactions that occur between oHAdVs and the host, make it particularly challenging to develop relevant animal models to investigate acute toxicity, immunity, efficacy, and pharmacodynamics for pre-clinical development of these agents. Here, the experimental options for pre-clinical evaluation of therapeutic oHAdV-based vectors for cancer therapy are reviewed, with a focus on Ad serotype 5 (Ad5), the most common serotype for therapeutic oHAdV-based vector development."

Journal • Preclinical • Review • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

Neoadjuvant Intravesical Nadofaragene Firadenovec With Gemcitabine, Cisplatin and Durvalumab for the Treatment of Muscle Invasive Bladder Cancer, TRIFECTA Trial (clinicaltrials.gov) - P2 | N=33 | Not yet recruiting | Sponsor: University of Washington

New P2 trial • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • Urothelial Cancer

Evolving Pharmacotherapy for Bladder Cancer-From Non-Muscle-Invasive to Metastatic Disease-Development of Novel Therapies for BCG-Unresponsive NMIBC (PubMed, Gan To Kagaku Ryoho) - "Bladder cancer is a common malignancy in the elderly, with approximately 75% of cases diagnosed as non-muscle-invasive bladder cancer(NMIBC).Although transurethral resection of bladder tumor(TURBT)is the standard initial treatment, recurrence rates remain high, and intravesical Bacillus Calmette-Guérin(BCG)instillation has long been used as an adjuvant therapy.However, about 30% of patients develop BCG-unresponsive disease despite adequate BCG administration.Radical cystectomy is recommended in such cases, but its invasiveness and impact on quality of life(QOL)often make it impractical in real-world settings.Thus, the development of novel bladder-preserving treatments is urgently needed.In recent years, diverse approaches including immune checkpoint inhibitors(ICI), gene therapy, drug delivery systems(DDS), and oncolytic virus-based therapies have been actively investigated, with numerous clinical trials ongoing worldwide.Importantly, pembrolizumab, nadofaragene..."

Journal • Bladder Cancer • Gene Therapies • Genito-urinary Cancer • Oncology • Solid Tumor

ABLE-22: Safety and efficacy evaluation of nadofaragene firadenovec alone or in combination with chemotherapy or immunotherapy—A randomized, open-label, phase 2 study. (ASCO-GU 2025) - P2 | "Bladder-preserving treatment options for patients with BCG-unresponsive NMIBC with CIS ± Ta/T1 include intravesical gene therapy (nadofaragene firadenovec-vncg), intravesical chemotherapy (gemcitabine and docetaxel), and immunotherapy (intravenous pembrolizumab and intravesical nogapendekin alfa inbakicept-pmln). Exploratory endpoints include changes in expression of potential biomarkers in blood and urine. Results from this investigational, randomized, multicenter, open-label study evaluating the safety and efficacy of nadofaragene firadenovec alone or in combination with chemotherapy or immunotherapy are expected July 2028."

Clinical • Combination therapy • IO biomarker • P2 data • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

A national perspective on the management of high-risk BCG-unresponsive non-muscle-invasive bladder cancer in Romania. (PubMed, Arch Ital Urol Androl) - "Though RC remains the predominant approach for BCG-unresponsive cases, over half of urologists' report using intravesical chemotherapy, reflecting interest in bladder-sparing strategies rather than newly approved FDA agents."

Journal • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

ABLE-32: A randomized, controlled, phase 3b clinical trial of nadofaragene firadenovec-vncg versus observation in patients with intermediate-risk non–muscle-invasive bladder cancer. (ASCO-GU 2025) - P3 | "Exploratory endpoints include effect on potential biomarkers and health-related quality of life. Final results are expected in 2031."

Clinical • P3 data • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor