Swiss biopharmaceutical company Ferring Pharmaceuticals has received approval for its Adstiladrin (nadofaragene firadenovec-vncg) gene therapy from the US Food and Drug Administration (FDA) for the treatment of a type of bladder cancer in adult patients.

Adstiladrin, which is an adenovirus vector-based gene therapy, is indicated for treating high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) irrespective of papillary tumors.

Adstiladrin FDA approval was backed by the findings of a phase 3 multicenter clinical study in 157 patients with high-risk BCG-unresponsive NMIBC. The late-stage trial showed that more than half of patients having carcinoma in situ with or without concomitant high-grade Ta or T1 disease achieved a complete response (CR) by three months and of the patients who achieved an initial CR, 46% continued to stay free of high-grade recurrence at 12 months.

Adstiladrin is said to offer NMIBC patients an alternative compared to an invasive bladder removal surgery.

Armin Metzger — Ferring Pharmaceuticals Executive Vice President and Chief Science Officer, commenting on Adstiladrin FDA approval, said: “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.

“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.”

Administered by catheter into the bladder once every three months, intravesical therapy Adstiladrin targets the patient’s own bladder wall cells to increase the body’s natural defence mechanism to fight cancer.

Adstiladrin is anticipated to be commercially available in the US in the second half of 2023.

With ongoing long-term follow-up phase of the four-year study, the patients are continuing to be monitored for five years.

In the US, approximately 57,000 men and 18,000 women are diagnosed with bladder cancer every year, and around 12,000 men and 4,700 women die from the disease annually in the country, according to the Centers for Disease Control and Prevention.

Recently, Ferring Pharmaceuticals secured Rebyota FDA approval for the prevention of the recurrence of Clostridioides difficile infection (CDI).

  Adstiladrin, Armin Metzger, bladder cancer, Ferring Pharmaceuticals, nadofaragene firadenovec, NMIBC, non-muscle invasive bladder cancer, US FDA, US Food and Drug Administration