Community practice experience shows early efficacy, favorable safety with nadofaragene

New real-world evidence (RWE) from a private clinical practice add to the growing body of evidence supporting the safety and efficacy of nadofaragene firadenovec-vncg (Adstiladrin) for patients with high-risk BCG-unresponsive non–muscle invasive bladder cancer (NMIBC) 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 in Chicago, Illinois.^1,2

Nadofaragene is an intravesical non-replicating adenoviral vector-based gene therapy that received FDA approval in December 2022 for this patient population. The treatment is administered locally as a monotherapy directly into the bladder via a catheter every 3 months.

In the pivotal trial (NCT02773849) that supported FDA approval, nadofaragene demonstrated an overall complete response (CR) rate of 51%, including a CR of 72.9% in patients with papillary tumors only. Additional data from a separate RWE analysis from the Mayo Clinic reported a 79% complete response rate.

In a recent interview with Urology Times®, John Eifler, MD, a urologic oncologist at First Urology in Louisville, Kentucky, outlined key findings and takeaways from an additional RWE study conducted at a private clinical practice. In total, the study included 13 patients with a median age of 77.7 years (range, 69 to 85). All patients were pretreated with 75 mg oral vibegron (Gemtesa), which is a medication used to manage overactive bladder.

Urology Times: Could you describe the background/rationale for this work?

Eifler: Nadofaragene is a gene therapy that is [indicated] for BCG-unresponsive non–muscle invasive bladder cancer with carcinoma in situ. These are patients who have high-grade, aggressive disease who have failed BCG in the past.

If you look at the trials and the studies that have been published previously for nadofaragene, there's the pivotal US phase 3 study that was initially presented. That demonstrated that about [51%] of patients who received nadofaragene had a complete response at 3 months. On the other hand, when the Mayo Clinic presented their real-world evidence, they saw a much higher complete response rate at 3 months, at 79%. We wanted to add to this body of literature with our early evidence in a real-world private practice setting.

Urology Times: What were the key results from this real-world analysis?

Eifler: When we looked at our data, we found that about 77% of patients were responders at 3 months. That included some patients who had only CIS and other patients who had papillary disease. For the patients with CIS, 70% of patients had a complete response at 3 months, and 100% of patients with the high-grade papillary disease had a complete response at 3 months.

The other thing that I think bears mention about our experience is that in our population, we used vibegron to help prevent bladder spasms. We have found this to be very effective. We did have 1 patient who did have expulsion of some of their treatment, and when we worked with the company, we were able to get an additional dose for the patient. She ended up being a complete responder, thankfully. After that, we did modify our protocol. We pre-treat with vibegron for a full week before they receive their dose of nadofaragene, and we also treat for a full week afterwards as well.

Urology Times: What did the safety data from this study show?

Eifler: In our experience, patients had very minor [adverse] effects, with the exception of bladder spasms at the time of the treatment. Patients had just grade 1 or grade 2 urgency and frequency. There were no grade 3, 4, or 5 adverse events.

Urology Times: In the context of these other studies of nadofaragene, what are the key takeaways for urologists?

Eifler: We found in our experience that the complete response rate for nadofaragene was 77%, which is comparable to the experience of the Mayo Clinic and considerably higher than the results published in the phase 3 study. So, we do find excellent complete response rates with nadofaragene.

There are a lot of great therapies that are coming out, and they all look like good options for patients with BCG-unresponsive, high-grade non–muscle invasive bladder cancer with CIS. I think nadofaragene is an excellent option that's well tolerated by patients, and at every 3 months instillation, I think it's very compelling.

REFERENCES

1. Malone RJ, Eifler JB, Shoskes DA. Real-world evidence of early efficacy for intravesical nadofaragene firadenovec in patients with high-risk non–muscle-invasive bladder cancer (NMIBC). Presented at: 99th Annual Meeting of the North Central Section of the American Urological Association. October 15 – 18, 2025. Chicago, Illinois. Abstract 163. https://ncsaua.org/docs/meetings/ncs2510/abstracts.aspx

2. Ferring expands growing body of real-world evidence reinforcing use of ADSTILADRIN® (nadofaragene firadenovec-vncg) in urology practices. News release. Ferring Pharmaceuticals. October 16, 2025. Accessed November 14, 2025. https://ferringusa.com/?press=ferring-expands-growing-body-of-real-world-evidence-reinforcing-use-of-adstiladrin-nadofaragene-firadenovec-vncg-in-urology-practices