ADSTILADRIN® Marks a New Era in Bladder Cancer Treatment with Robust Clinical and Real-World Data.

For over 30 years, Bacillus Calmette-Guérin (BCG) therapy has been the cornerstone in managing non-muscle invasive bladder cancer (NMIBC). Originally developed as a tuberculosis vaccine and approved by the U.S. FDA for bladder cancer in 1990, BCG has shown initial effectiveness. However, nearly one-third of patients fail to respond, and among responders, around 50% eventually face recurrence or progression of the disease. This has historically left patients with limited options—either undergo radical cystectomy, a life-altering surgery to remove the bladder, or risk disease progression to more dangerous stages.

While cystectomy can be curative, it is a highly invasive and permanent solution, often associated with considerable complications and diminished quality of life. According to market insights from Ferring Pharmaceuticals, nearly 95% of healthcare professionals prioritize treatment strategies that preserve the bladder without increasing the risk of cancer progression. Yet for decades, alternatives for BCG-unresponsive patients remained scarce.

This treatment landscape changed with the approval of ADSTILADRIN® (nadofaragene firadenovec), the first intravesical non-replicating gene therapy for BCG-unresponsive NMIBC with carcinoma in situ (CIS) with or without papillary tumors. Administered once every three months in an outpatient setting, ADSTILADRIN introduces a much-needed, bladder-sparing option that is both effective and manageable for patients.

The development of ADSTILADRIN spans more than 30 years and involved significant technical and regulatory breakthroughs. The first challenge was biological—interferon, known for its antitumor properties, failed to maintain sufficient therapeutic levels in the bladder due to its natural voiding function.

ADSTILADRIN overcame this by using a non-replicating adenovirus vector to deliver the gene for interferon alfa-2b directly into bladder cells. These cells then produce high, localized levels of the protein, transforming into temporary “interferon microfactories.” This method prolongs therapeutic exposure and triggers both direct tumor suppression and immune system activation

The second major hurdle was regulatory. For years, the medical community lacked a unified definition of BCG failure, making consistent clinical trial design difficult. In 2018, collaboration between the U.S. FDA and the uro-oncology community led to the formal classification of “BCG-unresponsive” NMIBC, providing the necessary criteria to advance clinical development. ADSTILADRIN received FDA approval in December 2022 for adult patients with high-risk BCG-unresponsive NMIBC with CIS with or without papillary tumors.

The pivotal Phase 3 trial conducted by the Society of Urologic Oncology Clinical Trials Consortium (SUO-CTC) demonstrated promising results: 51% of patients achieved a complete response (CR) at 3 months following a single treatment, and 46% of these responders remained disease-free at 12 months. Adverse events were typically mild to moderate, including instillation site discharge, fatigue, bladder spasms, urgency, and hematuria. Fewer than 2% of patients discontinued treatment due to side effects.

In January 2024, ADSTILADRIN became fully available in the U.S. Real-world evidence quickly followed. A retrospective Mayo Clinic study involving 46 patients confirmed its efficacy and tolerability. Among 24 evaluable patients, 79% achieved a CR at 3 months, with 84% of those maintaining their response over a median 7.3-month follow-up. These results mirrored those from the initial trials and were conducted independently of Ferring Pharmaceuticals.

Further global validation came from an ongoing Phase 3b trial in Japan, where 75% of patients reached a CR at 3 months. Drug-related side effects were mild (Grade 1 or 2), reinforcing the therapy’s strong safety profile. Importantly, no severe (Grade 4 or 5) adverse events have been reported in any clinical study to date.

Recognized by leading organizations such as the International Bladder Cancer Group (IBCG), the NCCN Guidelines, and AUA/SUO guidelines, ADSTILADRIN is emerging as a preferred treatment for BCG-unresponsive NMIBC. Ferring Pharmaceuticals continues to invest in additional clinical trials to expand its therapeutic potential and further cement its place as a foundational treatment in bladder cancer care.

Through innovative science and an enduring commitment to the bladder cancer community, ADSTILADRIN offers new hope—delivering on the promise of preserving quality of life while effectively managing a historically challenging disease.

Source:https://endpts.com/sp/adstiladrin-nadofaragene-firadenovec-vncg-a-growing-body-of-clinical-data-and-real-world-evidence/

This is non-financial/medical advice and made using AI so could be wrong.