FDA Approves First Gene Therapy for the Treatment of High-Risk, Non-Muscle-Invasive Bladder Cancer

  • Ferring’s novel adenovirus vector-based gene therapy Adstiladrin® (nadofaragene firadenovec-vncg) is the first gene therapy approved for bladder cancer
  • Efficacy and safety of Adstiladrin supported by Phase 3 results demonstrating that more than half of patients (51% of CIS ± Ta/T1 cohort) achieved a complete response (CR) at three months and of these, 46% continued to remain free of high-grade recurrence at 12 months
  • Bladder cancer is the sixth most common cancer in the U.S.; Adstiladrin provides NMIBC patients a valuable alternative compared to an invasive bladder removal surgery
Reno, Nevada (UroToday.com) -- Ferring Pharmaceuticals today announced the U.S. Food and Drug Administration (FDA) approved Adstiladrin® (nadofaragene firadenovec-vncg), a novel adenovirus vector-based gene therapy, for the treatment of adult patients with high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.

“Patients with BCG-unresponsive NMIBC have historically had limited treatment options other than bladder removal surgery,” said Steven A. Boorjian, M.D., Carl Rosen Professor and Chair of the Department of Urology at Mayo Clinic, and lead investigator on the recent clinical trial of Adstiladrin. “The approval of Adstiladrin is therefore a significant advance in the current treatment landscape and provides a novel treatment option for patients.”
Bladder cancer is the sixth most common cancer in the U.S., with NMIBC representing approximately 75% of all new bladder cancer cases.1,2 BCG remains the first-line standard of care for people living with high-grade NMIBC. However, more than 50% of patients who receive initial treatment with BCG will experience disease recurrence and progression within one year, with many developing BCG-unresponsive disease.3

Adstiladrin, an intravesical therapy administered every three months, targets the patient’s own bladder wall cells to enhance the body’s natural defenses to fight cancer. The FDA approval was based on results of the Phase 3 clinical trial, which met its primary endpoint with more than half (51%, n=50 of 98; 95% CI 41 to 61) of patients with carcinoma in situ with or without concomitant high-grade Ta or T1 disease (CIS ± Ta/T1) achieving a complete response (CR) by three months. Of the patients who achieved an initial CR, 46% (n=23 of 50) continued to remain free of high-grade recurrence at 12 months.

The approval of Adstiladrin showcases the power of private industry-academia partnerships in bringing novel treatments to market,” said Colin Dinney, M.D., Chairman, Department of Urology, Division of Surgery, University of Texas MD Anderson Cancer Center. “The Society of Urologic Oncology Clinical Trials Consortium (SUO-CTC) defined the clinical trial design required to address this patient population and has been a proud collaborator in the research of Adstiladrin, and we are delighted that such a transformative treatment is now approved by the FDA.”
“Ferring has been working diligently to realize the potential of gene therapy for bladder cancer patients, where there has long been a critical unmet need for additional treatment options,” said Armin Metzger, Executive Vice President and Chief Science Officer, Ferring Pharmaceuticals. “We are proud to have achieved this critical milestone towards fulfilling the potential of Adstiladrin, a first-of-its-kind therapy, for bladder cancer patients. Adstiladrin is the culmination of a complex research, development, and production process, and we are grateful to the teams, physicians and patients who have helped us reach this approval.”

Ferring expects that Adstiladrin will be commercially available in the United States in the second half of 2023, following manufacturing capacity expansion which will see the company pioneering commercial scale vector production for oncology.

For full prescribing information, please visit: https://www.ferringusa.com/wp-content/uploads/sites/12/2022/12/ADSTILADRIN_pi.pdf

This application was granted Priority ReviewBreakthrough Therapy, and Fast Track designations.

The FDA granted approval of Adstiladrin to Ferring Pharmaceuticals A/S.

Source: Ferring Pharmaceuticals, December 16, 2022, Ferring Receives Approval from U.S. FDA for Adstiladrin for High-Risk, BCG-Unresponsive Non-Muscle Invasive Bladder Cancer [Press Release]