The Food and Drug Administration approved Adstiladrin on Friday, the first gene therapy for a type of bladder cancer. Ferring Pharmaceuticals, the Swiss drugmaker behind it, said the product is for adults with an aggressive form of the disease. Until now, the options for those patients have included having the bladder removed.
The drug is given once every three months, by catheter, straight into the bladder. It is a non-replicating adenovirus vector carrying the gene for interferon alfa-2b, and it is aimed at adults with non-muscle invasive bladder cancer (NMIBC) that no longer responds to BCG. The vector enters cells of the bladder wall and releases the gene. The cells’ own machinery reads it and begins secreting large amounts of interferon alfa-2b, a protein the body naturally uses against cancer. In effect, the patient’s bladder cells become what the company’s description calls “interferon microfactories.”
Who it is for
NMIBC is the most common type of bladder cancer, and about 75% of all bladder cancer cases in the United States fall into it. It is usually found early and stays inside the bladder, without reaching the muscle wall or other parts of the body. Surgery comes first, then immunotherapy. Patients with high-grade papillary cancers or carcinoma in situ typically get BCG, a bacteria-based immunotherapy. Many still see the cancer progress. Beyond complete bladder removal, few alternatives exist.
The FDA said safety and effectiveness were evaluated in a multi-center clinical study of 157 patients.
Price and availability
Ferring said it was too early to discuss a price but that it aims to make the drug widely available to patients. A 2021 report from the Institute for Clinical and Economic Review, a drug pricing research group, concluded the drug offers good value at a price between $158,600 and $262,000. Ferring expects Adstiladrin to reach the U.S. market in the second half of 2023.
At UC San Diego, urologists have started bladder gene treatments on five patients, according to CancerConnect. The treatment could delay or remove the need for complete bladder removal if it works as hoped.
Sources
- Adstiladrin: US approves first gene therapy for bladder cancer
- New Gene Therapy for Bladder Cancer
- Cancer treatment
