Gene Therapy for Bladder Cancer Now Fully Available

Adstiladrin (nadofaragene firadenovec-vncg) is now fully available across the United States to treat adult patients with high-risk non-muscle-invasive bladder cancer (NMIBC) with carcinoma in situ with or without papillary tumors. It is indicated for patients who are resistant to Bacillus Calmette-Guérin (BCG), an immunotherapy that is used to treat patients with early-stage bladder cancer.

Developed Ferring Pharmaceuticals, Adstiladrin is the first intravesical gene therapy for adults with NMIBC who no longer respond to standard therapy. It was approved in December 2022 and is an adenoviral vector‑based gene therapy containing the gene interferon alfa-2b, administered by catheter into the bladder once every three months. This results in cells secreting high quantities of interferon alfa-2b, a naturally occurring protein the body uses to fight cancer.

Ferring listed Adstriladrin at $60,000 per unit dose. Each unit of is provided in a carton containing four vials. Each vial contains an extractable volume of not less than 20 mL. Ferring provides a copay assistance program for commercially insured patients where eligible patients pay as little as $100 for each prescription. The program, however, does not cover the cost of administration.

In September 2023, Ferring initiated the Adstiladrin Early Experience Program to clinical trial sites that participated in the phase 3 study, as well as some community clinics, to provide temporary access as supply was scaled up. Now that full product supply is available, Ferring will end the early experience program.

Ferring has initiated a real-world study to assess early utilization and outcomes. This observational study will enroll 800 patients and is expected to be completed by December 2026.

Bladder cancer is the sixth most common cancer in the United States. It is estimated that there were about 81,180 new cases of bladder cancer in the United States in 2022. Of these, 75% present as non-muscle-invasive bladder cancer, a cancer that is present in the superficial layer of the bladder and has not invaded the bladder itself. Bacillus Calmette-Guérin is standard of care, but more than half of patients will experience recurrence of cancer within one year.

New interim data from a 36-month follow-up analysis from the phase 3 study, one-quarter of patients with high-risk, BCG-unresponsive NMIBC who were treated with Adstiladrin remained free of high-grade recurrence. The study is ongoing with a planned five-year treatment and monitoring phase. These data were presented at the 24th Annual Meeting of the Society of Urologic Oncology in November 2023.

According to the prescribing information, the most common adverse events were increased glucose levels, instillation site discharge, an increase in triglycerides, fatigue, bladder spasm, urination urgency, increase in creatinine, blood in urine, phosphate decreased, chills, fever, and painful urination.