Novel intravesical gene therapy for NMIBC launches in select US clinics

The first patient has been dosed in a commercial setting with nadofaragene firadenovec-vncg (Adstiladrin), an intravesical gene therapy for the treatment of patients with BCG-unresponsive non–muscle-invasive bladder cancer (NMIBC).¹

“Adstiladrin represents a major advancement in the current treatment landscape for people living with high-risk NMIBC who may be facing removal of the bladder,” says Elizabeth Garner, MD, MPH.

According to Ferring Pharmaceuticals,¹ the developer of the gene therapy, the patient underwent treatment with nadofaragene firadenovec-vncg as part of the company’s Adstiladrin Early Experience Program. The program, which was announced earlier this year,² is initially available to clinical trial sites participating in the phase 3 study of the therapy as well as several community clinical sites with a high number of eligible patients with NMIBC.

Urologists at clinics participating in the Early Experience Program can now prescribe the gene therapy to eligible patients with NMIBC as well.

“Adstiladrin represents a major advancement in the current treatment landscape for people living with high-risk NMIBC who may be facing removal of the bladder,” Elizabeth Garner, MD, MPH, Chief Scientific Officer at Ferring Pharmaceuticals, USA, said in a news release.¹ “The Adstiladrin Early Experience Program and ABLE-41 US RWE Study allow us to address pressing patient needs while collecting data on its use in a real-world setting, further expanding on what we’ve learned in our Phase 3 clinical program.”

Nadofaragene firadenovec-vncg was approved by the FDA in December 2022 and is indicated for adult patients with high-risk BCG-unresponsive NMIBC with carcinoma in situ (CIS) with or without papillary tumors.

The therapy will be further assessed in the prospective ABLE-41 US real-world evidence (RWE) study (NCT06026332),³ which will explore the early utilization, experiences, and outcomes of the gene therapy in a routine care setting. The prospective trial plans to enroll up to 800 patients through the Early Experience Program who have not yet received nadofaragene firadenovec-vncg. Minimum follow-up of patients is 24 months.

The primary outcome measure is the complete response rate, as assessed at 3 months and 1 year. Secondary outcome measures include the duration of complete response, the rate of high-grade recurrence-free survival, progression-free survival, and overall survival.

“The first patient dosed with Adstiladrin marks an incredible milestone for Ferring, the bladder cancer community, and the patients we aim to serve. Making this novel and efficacious intravesical gene therapy commercially available for patients underscores our commitment to changing the trajectory of NMIBC. We will continue bringing available doses of Adstiladrin forward responsibly as we scale up manufacturing,” Shetal Vyas, the Vice President and General Manager of Uro-Oncology at Ferring Pharmaceuticals, said in a news release.¹

The Early Experience Program is expected to be offered to more clinics as manufacturing volumes of nadofaragene firadenovec-vncg increase.

References

1. First bladder cancer patient dosed with commercially available intravesical gene therapy ADSTILADRIN (nadofaragene firadenovec-vncg). News release. Ferring Pharmaceuticals. Published online and accessed September 12, 2023. https://www.businesswire.com/news/home/20230912802578/en/First-Bladder-Cancer-Patient-Dosed-with-Commercially-Available-Intravesical-Gene-Therapy-ADSTILADRIN%C2%AE-nadofaragene-firadenovec-vncg

2. Ferring Pharmaceuticals on track to deliver ADSTILADRIN (nadofaragene firadenovec-vncg) to patients in Early Experience Program in September 2023. News release. Ferring Pharmaceuticals. June 26, 2023. Accessed September 12, 2023. https://ferringusa.com/?press=ferring-pharmaceuticals-on-track-to-deliver-adstiladrin-nadofaragene-firadenovec-vncg-to-patients-in-early-experience-program-in-september-2023

3. National Institutes of Health US National Library of Medicine ClinicalTrials.gov. ADSTILADRIN early utilization and outcomes in the real world setting in the United States (ABLE-41). Last updated September 7, 2023. Accessed September 12, 2023. https://www.clinicaltrials.gov/study/NCT06026332