Ferring Pharmaceuticals has announced additional real-world evidence from a private urology practice evaluating the efficacy and safety of ADSTILADRIN® (nadofaragene firadenovec-vncg) in patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC), specifically those with carcinoma in situ (CIS) with or without papillary tumors (±Ta/T1). The findings were presented at the 99th Annual Meeting of the North Central Section of the American Urological Association (AUA) in Chicago, Illinois.
The study demonstrated a complete response (CR) rate of 77% (10 of 13 patients) at three months, which is particularly notable in this challenging patient population. Breaking down the outcomes, 70% of patients with CIS± papillary tumors (7 of 10) achieved a complete response, while all patients with papillary-only lesions (3 of 3) responded to treatment. These results highlight the potential of ADSTILADRIN as a highly effective therapy in a population with limited bladder-sparing options.
The patient cohort included thirteen individuals who met the study’s entry criteria, with a mean age of 77.7 years. Pretreatment pathology revealed 10 patients with CIS± papillary tumors and 3 patients with papillary-only lesions. Prior to enrollment, patients had received a median of 12 doses of BCG therapy (ranging from 6 to 19 doses), reflecting a heavily pretreated population with limited alternative therapies.
ADSTILADRIN was administered intravesically at a dose of 75 mL containing 3×10¹¹ viral particles/mL and retained in the bladder for one hour. To optimize tolerability and enhance patient comfort, all participants were premedicated with oral vibegron 75 mg, a therapy commonly used for overactive bladder. Impressively, nearly all patients (12 of 13) retained the therapy for the full one-hour dwell time, supporting the feasibility of administration in real-world settings.
Commenting on the findings, John Eifler, M.D., a urologist at First Urology in Louisville, KY, emphasized the importance of understanding real-world outcomes:
“The Phase 3 ADSTILADRIN research provides strong evidence for its efficacy and safety, but urologists and their patients also want to understand how these results translate to routine clinical practice. In our real-world study, we observed a three-month efficacy rate that exceeded the Phase 3 findings, underscoring the role of ADSTILADRIN as an effective and well-tolerated option for BCG-unresponsive patients.”
This perspective aligns with a growing emphasis in oncology on translating clinical trial success into real-world applicability, where patient populations may be older, more comorbid, or have received extensive prior therapies. Real-world studies provide additional context, helping clinicians make informed treatment decisions for patients who may not fit the stringent criteria of clinical trials.
Supporting this view, Daniel A. Shoskes, M.D., FRCS(C), Vice President and Global Medical Director-Uro-Oncology at Ferring Pharmaceuticals, noted:
“These new data add to the growing body of real-world evidence in urology practices offering patients a bladder-sparing, non-chemotherapy option after BCG fails. Real-world research, including studies supported by Ferring, helps to reinforce the efficacy and safety of ADSTILADRIN demonstrated in prospective trials and provides uro-oncologists the information needed to guide critical treatment decisions.”
ADSTILADRIN represents a novel gene therapy approach for high-risk NMIBC, designed to offer patients a bladder-sparing alternative when standard BCG therapy is no longer effective. The therapy’s mechanism involves intravesical delivery of a replication-deficient adenovirus vector, encoding the interferon alpha-2b gene, which induces a targeted antitumor immune response within the bladder lining.
With the increasing prevalence of NMIBC and the limitations of repeated BCG therapy, these real-world findings are particularly encouraging. They highlight the potential for ADSTILADRIN to fill a critical therapeutic gap for patients who might otherwise face radical cystectomy or systemic chemotherapy.
As clinical adoption grows, additional studies and ongoing real-world experience will continue to inform best practices, helping to optimize patient outcomes and expand access to this innovative treatment option.
