The US Food and Drug Administration on Friday approved Adstiladrin, Swiss drugmaker Ferring Pharmaceuticals’ first gene therapy for treating adult patients with a type of bladder cancer.
The therapy, Adstiladrin, is for patients with an aggressive form of the disease whose current options include having their bladder removed. The treatment, to be administered once every three months into the patient’s bladder, triggers the body to make a protein to fight off cancer.
The safety and effectiveness of Adstiladrin was evaluated in a multi-center clinical study that included 157 patients, the FDA said.
Ferring said it was too early to comment on the pricing of Adstiladrin, but it is aiming to make the drug widely accessible to patients.
According to a 2021 report from drug pricing research group Institute for Clinical and Economic Review, the drug provides a good value for patients when it is priced in the range of $158,600-$262,000.
Adstiladrin is expected to be commercially available in the United States in the second half of 2023, Ferring said.
Bladder cancer is one of the most common cancer in the United States, with non-muscle invasive bladder cancer (NMIBC) representing about 75% of all such cases.
Adstiladrin is a gene therapy developed as a treatment for adult patients with BCG-unresponsive NMIBC. It is a non-replicating adenovirus vector-based gene therapy containing the gene interferon alfa-2b, administered by catheter into the bladder once every three months. The vector enters the cells of the bladder wall, releasing the active gene to do its work.
The internal gene/DNA machinery of the cells “picks up” the gene and translates its DNA sequence, resulting in the cells secreting high quantities of interferon alfa-2b protein, a naturally occurring protein the body uses to fight cancer. This novel gene therapy approach thereby turns the patient’s own bladder wall cells into interferon microfactories, enhancing the body’s natural defenses against the cancer.