Ferring Pharmaceuticals has announced that the first patient in the United States suffering from high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) has been administered with the intravesical gene therapy ADSTILADRIN (nadofaragene firadenovec-vncg). The gene therapy was approved by the U.S. Food and Drug Administration (FDA) in December 2022 for the treatment of adult patients with this particular type of bladder cancer who are unresponsive to standard therapy.

Key Details of the Therapy and Program

ADSTILADRIN is part of Ferring Pharmaceuticals’ Early Experience Program that was introduced earlier this year. This therapy is the first and only FDA-approved intravesical gene therapy for adults with high-risk, BCG-unresponsive NMIBC. The treated patient, a 78-year-old male, is also enrolled in a research study known as the “ADSTILADRIN in BLadder CancEr” (ABLE-41) U.S. Real World Evidence (RWE) Study, aiming to assess the early utilization and outcomes of this gene therapy.

Industry and Patient Implications

“The first patient dosed with ADSTILADRIN marks an incredible milestone for Ferring, the bladder cancer community, and the patients we aim to serve,” said Shetal Vyas, Vice President, General Manager, Uro-Oncology at Ferring Pharmaceuticals. The therapy and accompanying study aim to meet pressing patient needs while collecting real-world evidence. Elizabeth Garner, MD, MPH, Chief Scientific Officer at Ferring Pharmaceuticals, remarked, “ADSTILADRIN represents a major advancement in the current treatment landscape for people living with high-risk NMIBC.”

Future Availability and Research

Earlier this month, Ferring Pharmaceuticals made doses of ADSTILADRIN available commercially through its Early Experience Program. The program, which also includes urologists who participated in the Phase 3 study, seeks to ensure continued future doses for patients currently enrolled. The ABLE-41 U.S. RWE Study, running parallel to the Early Experience Program, aims to collect data on the early utilization and experiences of this ground-breaking therapy.

  Adstiladrin, bladder cancer, Elizabeth Garner, Ferring Pharmaceuticals, USA