FDA Approves First Gene Therapy for Non-Muscle Invasive Bladder Cancer

Adstiladrin represents the first gene therapy approved by the FDA for the treatment of patients with high-risk non-muscle invasive bladder cancer who are resistant to BCG, the first-line standard of care for these patients.

The Food and Drug Administration (FDA) approved Adstiladrin (nadofaragene firadenovec-vncg) for the treatment of adults with high-risk, non-muscle invasive bladder cancer, representing the first gene therapy approved for this patient population.

In particular, Adstiladrin had been approved for the treatment of high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer with carcinoma in situ (a condition in which abnormal cells resembling cancer cells are found in the location where they first formed and have not spread to nearby tissue) with or without papillary tumors (tumors that look like long, thin lines that grow inside an organ).

“This approval provides health care professionals with an innovative treatment option for patients with high-risk, non-muscle invasive bladder cancer that is unresponsive to BCG therapy,” said Dr. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, in a release from the Agency. “Today’s action addresses an area of critical need.”

This FDA approval was based on findings from a phase 3 clinical trial assessing the safety and effectiveness of Adstiladrin, according to the FDA’s release. In this trial, 157 patients with high-risk BCG-unresponsive non-muscle invasive bladder cancer were treated with Adstiladrin once every three months for up to 12 months, or until unacceptable toxicity or disease recurrence. Of these patients, 51% achieved a complete response (disappearance of all signs of cancer as determined by biopsied tissue, cystoscopy and urine). The median duration of response (time from treatment to disease progression or death) to Adstiladrin was 9.7 months, and 46% of patients who responded to treatment remained in complete response for at least one year.

“Patients with BCG-unresponsive (non-muscle invasive bladder cancer) have historically had limited treatment options other than bladder removal surgery,” said Dr. Steven A. Boorjian, Carl Rosen Professor and Chair of the Department of Urology at Mayo Clinic and lead investigator of the trial, in a press release from Ferring Pharmaceuticals, the manufacturer of the therapy. “The approval of Adstiladrin is therefore a significant advance in the current treatment landscape and provides a novel treatment option for patients.”

The gene therapy Adstiladrin is administered into the bladder by a urinary catheter once every three months, according to the FDA’s release. The most common side effects from the therapy included fatigue, bladder discharge, bladder spasm, blood in the urine, urinary urgency, fever, chills and painful urination. Of note, patients who are immune deficient should not come into contact with Adstiladrin, according to the FDA.

Adstiladrin is expected to be commercially available in the U.S. in the second half of 2023, according to the company’s release.

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