FDA approves first gene therapy for high-risk early bladder cancer

The US Food and Drug Administration (FDA) has approved Adstiladrin (nadofaragene firadenovec-vncg) as the first gene therapy for non-muscle-invasive bladder cancer (NMIBC) in adults with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive with carcinoma in situ (CIS) with or without papillary tumours.

Dr Peter Marks, PhD, Director of the FDA’s Center for Biologics Evaluation and Research observed that Adstiladrin, the non-replicating adenoviral vector-based therapy provides “… an innovative treatment option for patients with high-risk non-muscle-invasive bladder cancer that is unresponsive to BCG therapy.”

Treatment and care of patients with high-risk NMIBC, including those with CIS often involves removing the tumour and the use of the BCG vaccine to reduce the risk that the cancer will recur. Few effective treatment options exist for patients who develop BCG-unresponsive disease.

The failure to achieve a complete response (CR), or the disappearance of all signs of cancer as seen on cystoscopy, biopsied tissue, and urine, is associated with an increased risk of death or a disease-worsening event. Without treatment, the cancer can invade, damage tissues and organs, and spread through the body.

Clinical study of Adstiladrin

The safety and effectiveness of Adstiladrin was evaluated in a multicentre clinical study that included 157 patients with high-risk BCG-unresponsive NMIBC, 98 of whom had BCG-unresponsive CIS with or without papillary tumours and could be evaluated for response.

Patients received Adstiladrin once every three months via a urinary catheter into the bladder for up to 12 months, or until unacceptable toxicity to therapy or recurrent high-grade NMIBC. Overall, 51 percent of patients achieved a CR. The median duration of response was 9.7 months. The percentage of responding patients who remained in CR for at least one year was forty-six percent.

The most common adverse reactions (ARs) included bladder discharge, fatigue, bladder spasm, urinary urgency, haematuria (presence of blood in urine), chills, fever, and painful urination. The FDA stated that individuals who are immunosuppressed, or immune-deficient should not come into contact with Adstiladrin.

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

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