FDA Approves First Gene Therapy For High-Risk, Non-Muscle-Invasive Bladder Cancer

The U.S. Food and Drug Administration has approved Ferring Pharmaceuticals A/S’s Adstiladrin (nadofaragene firadenovec-vncg) to treat high-risk, non-muscle-invasive bladder cancer.

Adstiladrin is a non-replicating, i.e., cannot multiply in human cells, adenoviral vector based gene therapy indicated 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.

It is the first gene therapy to be approved by the FDA to treat such high-risk, non-muscle-invasive bladder cancer, which is a disease in which malignant cells form a tumor in the tissues of the bladder.

According to the Centers for Disease Control and Prevention, about 57,000 men and 18,000 women are diagnosed with bladder cancer annually in the United States.

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

In the trial, patients received Adstiladrin once every three months for up to 12 months, or until unacceptable toxicity to therapy or recurrent high-grade NMIBC.

Adstiladrin is administered once every three months into the bladder through a urinary catheter. Individuals who are immunosuppressed, or immune-deficient should not come into contact with Adstiladrin.

The most common adverse reactions associated with Adstiladrin included bladder discharge, fatigue, bladder spasm, urinary urgency, hematuria (presence of blood in urine), chills, fever, and painful urination.

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

Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, “This approval provides healthcare professionals with an innovative treatment option for patients with high-risk non-muscle invasive bladder cancer that is unresponsive to BCG therapy.”

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