First Patient Receives Gene Therapy for BCG-Unresponsive Non-Muscle Invasive Bladder Cancer


Adstiladrin was approved in December 2022 and is now available for select patients under an Early Experience Program.

The first patient with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) has been treated with nadofaragene firadenovec (Adstiladrin; Ferring Pharmaceuticals) as part of an Early Experience Program.1

“The first patient dosed with Adstiladrin marks an incredible milestone for Ferring, the bladder cancer community and the patients we aim to serve,” Shetal Vyas, vice president, general manager, uro-oncology, Ferring Pharmaceuticals, said in a statement.1 “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.”

Nadofaragene firadenovec was approved by the FDA in December 2022 for treating adult patients with high-risk, BCG-unresponsive NMIBC with carcinoma in situ with or without papillary tumors.2 The FDA previously issued a complete response letter to Ferring in 2021 due to problems with chemistry, manufacturing, and controls.

The therapy, a nonreplicating adenoviral vector-based gene therapy, is administered intravesically every 3 months. It was developed as part of a collaboration between Ferring and the Society of Urologic Oncology Clinical Trials Consortium, who defined the clinical trial design.

READ MORE: NMI Bladder Cancer Gene Therapy Shows Promising Safety and Efficacy in Phase 1 Trial

Nadofaragene firadenovec was evaluated in 157 participants in an open-label phase 3 clinical trial (NCT02773849). The trial’s primary end point was complete response rate at 12 months, which was achieved by more than half of patients treated (51%; 95% CI, 41-61) by 3 months. Of these patients, 46% remained free of high-grade recurrence at 12 months (median duration, 9.7 months). The most common adverse events (AEs) in the trial included bladder discharge, fatigue, bladder spasm, urinary urgency, hematuria, chills, fever, and pain while urinating. Just under 2% of patients discontinued treatment due to AEs.

The treated patient is a 78-year-old man treated at a clinic within the mid-west area and was also enrolled in the “ADSTILADRIN in BLadder CancEr” (ABLE-41) United States Real World Evidence (RWE) study (NCT06026332), which Ferring has launched in tandem with the Early Experience program.

“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, Ferring Pharmaceuticals, added.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.”

1. First bladder cancer patient dosed with commercially available intravesical gene therapy ADSTILADRIN® (nadofaragene firadenovec-vncg). News release. September 12, 2023. Ferring Pharmaceuticals.
2. FDA Approves First Gene Therapy for the Treatment of High-Risk, Non-Muscle-Invasive Bladder Cancer. News release. FDA. December 16, 2022.
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