FDA Approves First Gene Therapy for Bladder Cancer

M. Alexander Otto, PA, MMS

December 16, 2022

The US Food and Drug Administration has approved nadofaragene firadenovec-vncg (Adstiladrin), the first gene therapy for adults with bladder cancer.

The adenovirus vector based gene therapy is indicated for adults with high-risk non-muscle invasive bladder cancer with carcinoma in situ with or without papillary tumors who are unresponsive to Bacillus Calmette-Guérin (BCG) therapy.

Patients with BCG-unresponsive disease "have historically had limited treatment options other than bladder removal surgery. The approval of Adstiladrin is therefore a significant advance in the current treatment landscape and provides a novel treatment option," Steven Boorjian, MD, a Mayo Clinic urologist and lead investigator on that agent's approval trial, said in a press release today from gene therapy developer Ferring Pharmaceuticals.

Nadofaragene firadenovec-vncg is instilled into the bladder via urinary catheter once every 3 months for up to a year. The adenovirus vector enters the cells of the bladder wall, releasing a gene that directs the cells to secrete high quantities of interferon alfa-2b, a naturally occurring cancer-fighting protein.

Approval was based on a multicenter clinical study that included 98 evaluable patients with high-risk, BCG-unresponsive disease. Overall, 51% achieved a complete response with a disappearance of all signs of cancer on cystoscopy, biopsied tissue, and urine. The median duration of response was 9.7 months. Overall, 46% of responding patients remained in complete response for at least 1 year.

The most common adverse events were instillation site discharge (33%), fatigue (24%), bladder spasm (20%), micturition urgency (19%), and hematuria (17%). The discontinuation rate as a result of adverse events was 1.9%.

Ferring expects the gene therapy to be commercially available in the United States in the second half of 2023.

The cost of the gene therapy has not yet been announced, but a cost effectiveness analysis from the Institute for Clinical and Economic Review, published last year, put the range for the annual cost between $158,600 and $262,000.

M. Alexander Otto is a physician assistant with a master’s degree in medical science and a journalism degree from Newhouse. He is an award-winning medical journalist who worked for several major news outlets before joining Medscape. Alex is also an MIT Knight Science Journalism fellow. Email: aotto@mdedge.com.

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